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Antisense or RNA interference

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514 - Drug, bio-affecting and body treating compositions

514001000 - DESIGNATED ORGANIC ACTIVE INGREDIENT CONTAINING (DOAI)

514023000 - Carbohydrate (i.e., saccharide radical containing) DOAI

514042000 - N-glycoside

514043000 - Nitrogen containing hetero ring

514044000 - Polynucleotide (e.g., RNA, DNA, etc.)

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DocumentTitleDate
20080207541Compositions and Methods For Optimizing Cleavage of Rna By Rnase H - The present invention provides compositions and methods for the optimization of cleavage of RNA species by RNase H. In some embodiments, the invention provides oligonucleotides that possess two or more regions of differing conformation, and at least one transitional nucleobase positioned between the regions that is capable of modulating transfer of the helical conformation characteristic of the region bound to the 3′hydroxy thereof, to the region bound to the 5′ hydroxyl thereof.08-28-2008
20090029932Identification and use of miRNAs for differentiating myeloid leukemia cells - The invention relates to the use of nucleic acid miRNA derived molecules for producing a drug for treating a myelogenous leukemia and to a method for identifying therapeutic agents or the efficient combination thereof for inducing the differentiation of myelogenous leukemia cells.01-29-2009
20090137518Therapeutic Antiangiogenic Endostatin Compositions - Endostatin compositions capable of inhibiting endothelial cell proliferation, inhibiting angiogenesis and causing tumor regression are described. Specifically, amino acid sequences of endostatin proteins and nucleic acid sequences coding for endostatin proteins are provided.05-28-2009
20090137513RNA Interference Mediated Inhibition of Acetyl-CoA-Carboxylase Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating acetyl-CoA carboxylase gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of acetyl-CoA carboxylase gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of acetyl-CoA carboxylase genes.05-28-2009
20090156540METHOD FOR DIAGNOSING AND/OR PREDICTING THE DEVELOPMENT OF AN ALLERGIC DISORDER AND AGENTS FOR TREATING AND/OR PREVENTING SAME - The present invention relates to genes whose level of expression is different in allergic animals compared with non-allergic animals. In particular, the present invention relates to a method for predicting the development of an allergic disorder in a mammal by determining the gene expression pattern of a panel of specific sequences comprising CAMK2D and CDH1 within a nucleic acid pool that have been predetermined to either increase or decrease in response to allergy.06-18-2009
20090192118TAK1-D MEDIATED INDUCTION OF CELL DEATH IN HUMAN CANCER CELLS BY SPECIFIC SEQUENCE SHORT DOUBLE-STRANDED RNAS - The splicing variant D of the TAK1 gene is activated by short double-stranded RNAs in a sequence specific manner. Activation of TAK1-D leads to the downstream activation of the p38 MAPK and of SAPK/JNK but not the NFκB pathway. The current invention therefore provides a method of inducing apoptosis and/or cell cycle arrest in a cancer cell comprising contacting said cell with an agonist of Tak1-D function. The invention further provides a method of modulating inflammation and the treatment of cancer by the administration of an agonist of Tak1-D function or expression. In yet another aspect, the invention provides a method of inducing p38 MAPK and SAPK/JNK signaling in a cell comprising contacting said cell with an agonist of Tak1-D function or expression.07-30-2009
20090192117COMPOSITIONS AND METHODS FOR THE SUPPRESSION OF TARGET POLYNUCLEOTIDES FROM LYGUS - Methods and compositions are provided which employ a silencing element that, when ingested by a pest, such as a pest from the 07-30-2009
20090192115USE OF COMPOUNDS THAT INTERFERE WITH THE HEDGEHOG SIGNALING PATHWAY FOR THE MANUFACTURE OF A MEDICAMENT FOR PREVENTING, INHIBITING, AND/OR REVERSING OCULAR DISEASES RELATED WITH OCULAR NEOVASCULARIZATION - The use of compounds that interfere with the hedgehog signaling pathway for the manufacture of a medicament for preventing, inhibiting, and/or reversing ocular diseases related with ocular neovascularization. Particularly, the above-mentioned diseases are (wet) age-related macular degeneration, (proliferative) diabetic retinopathy, neovascular glaucoma, retinal vein occlusion, or retinopathy of prematurity (ROP).07-30-2009
20090192111miR-124 Regulated Genes and Pathways as Targets for Therapeutic Intervention - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-124, using miR-124 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.07-30-2009
20090192110RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF PIK3CA EXPRESSION - The present invention relates to oligomeric compounds (oligomers), which target PIK3CA mRNA in a cell, leading to reduced expression of PIK3CA. Reduction of PIK3CA expression is beneficial for the treatment of certain medical disorders, such as hyperproliferative diseases (e.g., cancer). The invention provides therapeutic compositions comprising oligomers and methods for modulating the expression of PIK3CA using said oligomers, including methods of treatment.07-30-2009
20090192108Gene overexpressed in cancer - Disclosed are a protein encoded by a gene having a nucleotide sequence represented by any of SEQ ID NOs: 1 to 65 or a fragment thereof, an antibody recognizing the protein or antigen-binding fragment thereof, and a polynucleotide having a sequence comprising at least 12 consecutive nucleotides of a nucleotide sequence represented by any of SEQ ID NOs: 1 to 65 or a nucleotide sequence complementary thereto. The gene and the protein of the invention is useful for diagnosing and treating cancer.07-30-2009
20090192107Breast Cancer Markers - A method for the detection of the presence of or the risk of cancer in a patient, comprising, with reference to a normal control, the step of: (i) detecting the expression level of a gene characterised by any of the nucleotide sequences identified herein as SEQ ID No. 1 to SEQ ID No. 10, in a sample isolated from a patient, wherein an increased expression level of the gene characterised by any of SEQ ID Nos. 1 to 6 and 10, or a decreased expression level of the gene characterised by any of SEQ ID Nos. 7 to 9, indicates the presence of or the risk of cancer in the patient from whom the sample was isolated.07-30-2009
20090192102miR-21 REGULATED GENES AND PATHWAYS AS TARGETS FOR THERAPEUTIC INTERVENTION - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-21, using miR-21 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.07-30-2009
20090192106MODULATION OF eIF4E EXPRESSION - Oligomeric compounds, compositions and methods are provided for modulating the expression of eIF4E. The antisense compounds may be single- or double-stranded and are targeted to nucleic acid encoding eIF4E. Methods of using these compounds for modulation of eIF4E expression and for diagnosis and treatment of diseases and conditions associated with expression of eIF4E are provided.07-30-2009
20090192105RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCELIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of ICAM gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of ICAM genes.07-30-2009
20090192104RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating hypoxia inducible factor (e.g., HIF1) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of HIF1 gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HIF1 genes.07-30-2009
20090192103Multitargeting Interfering RNAs Having Two Active Strands And Methods For Their Design And Use - Interfering RNA molecules are now designed and produced with specificity for multiple binding sequences present in distinct genetic contexts in one or more pre-selected target RNA molecules and are used to modulate expression of the target sequences. The multitargeting interfering RNA molecules have two strands that target multiple target sites on one or more pre-selected RNA molecules. Such a multitargeting interfering RNA approach provides a powerful tool for gene regulation.07-30-2009
20090192101CANCER-SPECIFIC PROMOTERS - The present invention regards cancer-specific control sequences that direct expression of a polynucleotide encoding a therapeutic gene product for treatment of the cancer. Specifically, the invention encompasses breast cancer-specific and ovarian cancer-specific control sequences. Two breast cancer-specific sequences utilize specific regions of fatty acid synthase and claudin 4 promoters, particularly in combination with a two-step transcription amplification sequence and/or a post-transcriptional control sequence. Two ovarian cancer-specific sequences utilize specific regions of hTERT and survivin promoters, particularly in combination with a two-step transcription amplification sequence and/or a post-transcriptional control sequence. In more particular embodiments, these polynucleotides are administered in combination with liposomes.07-30-2009
20080242629ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of apolipoprotein B. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding apolipoprotein B. Methods of using these compounds for modulation of apolipoprotein B expression and for treatment of diseases associated with expression of apolipoprotein B are provided.10-02-2008
20080318895INTRANASAL DELIVERY OF NUCLEIC ACID MOLECULES - Aerosol delivery of nucleic acids to the lungs using viral vectors, polymers, surfactants, or excipients has been described. Compositions for intranasal administration are described that contain nucleic acids without viral or plasmid vectors and with little to no polymers, surfactants, or excipients. In one embodiment, the composition for intranasal delivery consists essentially of at least one nucleic acid and an aqueous solution. Suitable nucleic acids for intranasal delivery include, but are not limited to, dsDNA, dsRNA, ssDNA, ssRNA, short interfering RNA, micro-RNA, and antisense RNA Methods for treatment, diagnosis, or prevention of at least one symptom or manifestation of a lung disease are also described consisting of administration by intranasal delivery an effective amount of a composition containing a nucleic acid. The composition may be formulated as a liquid or aerosol or other acceptable formulation for intranasal administration.12-25-2008
20090239815Novel human microRNAs associated with cancer - The invention provides new sequences for human microRNAs associated with cancer which may be used as molecular markers for cancer diagnostics or as therapeutic targets or agents.09-24-2009
20090118206RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS - The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.05-07-2009
20090075928G-QUARTET OLIGONUCLEOTIDES THAT TARGET HYPOXIA-INDUCIBLE FACTOR 1-a (HIF1a ) - The present invention concerns particular G-quartet oligonucleotides that are employed for the treatment and/or prevention of cancer. In specific cases, the G-quartet oligonucleotides inhibit HIF-1α.03-19-2009
20090088401In-situ cancer autovaccination with intratumoral stabilized dsRNA viral mimic - An improved autovaccination method designed to prevent or treat various neoplastic diseases by inducing a systemic immune response against a tumor and its remote metastases, consisting of the induction of an immunogenic cell death in one or more targeted tumor sites with local radiation therapy, cryotherapy, heat, chemotherapy or various other treatments, followed by intratumoral/peritumoral injection of dsRNAs (poly-ICLC in particular) in the same tumor site.04-02-2009
20090143321NUCLEIC ACID AGENTS FOR DOWNREGULATING H19 AND METHODS OF USING SAME - The present invention provides isolated oligonucleotides capable of down-regulating a level of H19 mRNA in cancer cells. Articles of manufacture comprising agents capable of downregulating H19 mRNA in combination with an additional anti-cancer treatment are disclosed as well as methods of treating cancer by administering same.06-04-2009
20090176728Treatment of CNS Conditions - Methods and compositions for the treatment of pathologic conditions of the central nervous system (CNS) by means of intranasal administration of a composition that modulates, by means of RNA interference, the expression and/or activity of genes involved in above-mentioned conditions.07-09-2009
20090099110Antiviral oligonucleotides - Random sequence oligonucleotides that have antiviral activity are described, along with their use as antiviral agents. In many cases, the oligonucleotides are greater than 40 nucleotides in length. Also described are methods for the prophylaxis or treatment of a viral infection in a human or animal, and a method for the prophylaxis treatment of cancer caused by oncoviruses in a human or animal. The methods typically involve administering to a human or animal in need of such treatment, a pharmacologically acceptable, therapeutically effective amount of at least oligonucleotide that does not act by a sequence complementary mode of action.04-16-2009
20090005335COMPOUNDS FOR THE MODULATION OF BETA-CATENIN EXPRESSION - The invention relates to oligomer compounds (oligomers), which target beta-catenin mRNA in a cell, leading to reduced expression of beta-catenin. Reduction of beta-catenin expression is beneficial for a range of medical disorders, such as hyperproliferative disorders, such as cancer. The invention provides therapeutic compositions comprising oligomers and methods for modulating the expression of beta-catenin using said oligomers, including methods of treatment.01-01-2009
20090163436METHODS FOR DELIVERY OF NUCLEIC ACIDS - This invention features methods and compositions for delivery of nucleic acids (e.g., DNA, RNA, PNA, and hybrids thereof) to cells. The nucleic acid delivery complexes of the invention permit biologically active nucleic acids to be delivered to cells and organisms in vitro and in vivo in a manner and form that allows the nucleic acids to carry out their desired biological function.06-25-2009
20090163435miR-200 REGULATED GENES AND PATHWAYS AS TARGETS FOR THERAPEUTIC INTERVENTION - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-200, using miR-200 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.06-25-2009
20090163434miR-20 Regulated Genes and Pathways as Targets for Therapeutic Intervention - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-20a, using miR-20a to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.06-25-2009
20090093424Markers for melanoma - A method of diagnosing melanoma and/or monitoring melanoma progression, in particular for determining whether the melanoma is likely to have metastatic capabilities, in a subject includes the steps of in a test sample determining the methylation status of FABP5 wherein methylation of the gene indicates a positive diagnosis of melanoma and/or increased methylation of the gene indicates the progression of melanoma. Methods of monitoring melanoma progression may also incorporate measuring the expression levels of FABP5, with low levels of expression indicating a positive diagnosis of melanoma and/or lower levels of expression being indicative of progression of melanoma. Methods of treating melanoma in a subject comprise administering a therapeutically effective amount of a DNA methyltransferase inhibitor or a histone deacetylase inhibitor to the subject such that expression of FABP5 is increased. Microarrays for use in these diagnostic methods and compound screening methods based around monitoring methylation and/or expression of FABP5 are also envisaged.04-09-2009
20090156543METHODS AND COMPOSITIONS RELATING TO EXPRESSION FACTORS - The invention describes an expression factor and methods for inhibiting the growth of cells, for enhancing the activity of a drug, and for inhibiting the virulence of microbes. Methods of screening for expression factor inhibitors are also described. The compositions comprise at least one expression factor inhibitor and may further comprise at least one drug.06-18-2009
20090156542MCP-1 Binding Nucleic Acids And Use Thereof - The present invention is related to a nucleic acid molecule capable of binding to MCP-1, whereby the nucleic acid molecule is for use as a medicament for the treatment and/or prevention of a chronic disease or chronic disorder, preferably selected from the group consisting of chronic respiratory disease, chronic kidney disease and systemic lupus erythematosus.06-18-2009
20090156541DBAIT AND USES THEREOF - The invention relates to compositions and methods for interfering with the DNA repair of double strand breaks (DSBs). The invention discloses novel double-stranded nucleic acid molecules. that act as baits and hijack the holocomplex of enzymes responsible of DNA DSB sensing, signaling and/or repair pathways, in particular the non homologous end joining (NHEJ) pathway of DSB repair.06-18-2009
20090156538MODULATION OF ENDOTHELIAL LIPASE EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of endothelial lipase. The compositions comprise oligonucleotides, targeted to nucleic acid encoding endothelial lipase. Methods of using these compounds for modulation of endothelial lipase expression and for diagnosis and treatment of disease associated with expression of endothelial lipase are provided.06-18-2009
20090156524Novel siRNAS and methods of use thereof - The invention relates to compounds, in particular siRNAs, which inhibit the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject. The invention also provides antibodies which inhibit specified human polypeptides and pharmaceutical compositions comprising one or more such antibodies.06-18-2009
20090156530Norepinepherine Transporter Mutants and Uses Thereof - The present invention provides norepinepherine transporter (NET) mutants which display altered phosphorylation at site T30 and altered receptor trafficking. Methods for the use of the NET mutants, e.g., screening of compounds which alter NET trafficking, are also provided. A transgenic animal such as a mouse may comprise a NET mutant of the present invention.06-18-2009
20090156529RNAi Inhibition of Alpha-ENaC Expression - The invention relates to compositions and methods for modulating the expression of alpha-ENaC, and more particularly to the downregulation of alpha-ENaC expression by chemically modified oligonucleotides.06-18-2009
20090156528RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention concerns methods and reagents useful in modulating hepatitis C virus (HCV) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against hepatitis C virus (HCV) gene expression and/or activity. The small nucleic acid molecules are useful in the treatment and diagnosis of HCV infection, liver failure, hepatocellular carcinoma, cirrhosis and any other disease or condition that responds to modulation of HCV expression or activity.06-18-2009
20090156537COMPOSITIONS AND METHODS FOR DIAGNOSING AND TREATING ASTHMA OR OTHER ALLERGIC OR INFLAMMATORY DISEASES - The present invention provides compositions and methods useful for detecting or treating asthma or other allergic or inflammatory diseases. In one aspect, the methods of the present invention include inhibiting the activity or expression of a component of an arginine metabolic pathway in tissues affected by asthma or other allergic or inflammatory diseases. In many embodiments, the component being inhibited is a cationic amino acid transporter, an arginase, or a component downstream of the arginase. In many other embodiments, the activity or expression of the component is inhibited by an agent that binds to the component. In still many other embodiments, the activity or expression of the component is inhibited by an agent that binds a polynucleotide encoding the component.06-18-2009
20090156533RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of stromal cell-derived factor-1 (SDF-1) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in SDF-1 gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating or that mediate RNA interference (RNAi) against SDF-1 gene expression. Such small nucleic acid molecules are useful, for example, in providing compositions for treatment of traits, diseases and conditions that can respond to modulation of SDF-1 expression in a subject, such as ocular disease, cancer and proliferative diseases and any other disease, condition, trait or indication that can respond to the level of SDF-1 in a cell or tissue.06-18-2009
20090156532SNP BINDING SITE FOR microRNAs IN HLA-G - Analysis of microRNA interference with HLA-G expression identified the HLA-G 3′UTR SNP+3142G/C that disrupts a target for microRNA 148 (miR148) and is associated with asthma. The polymorphism is associated with protection from (or susceptibility to) moderate to severe viral infection in the first 3 years of life and asthma by age 6, with an interaction with mother's affection status (asthma). A SNP in the 3′-Untranslated Region (UTR) of HLA-G influences the targeting of 3 micro(mi)RNAs to the gene. Allele-specific targeting of these miRNAs accounts, at least in part, for the association between HLA-G and the risk of asthma.06-18-2009
20090156526COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION - Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.06-18-2009
20090156525Low-Density Lipoprotein Receptor-Related Protein 2 Clears Amyloid-Beta Peptide A cross the Blood-Brain Barrier via Apolipoprotein J - Low-density lipoprotein receptor-related protein 2 (LRP2) is a potential receptor to regulate the level of apolipoprotein J (apoJ) in the central nervous system, which is the major carrier of amyloid-β peptide (Aβ). ApoJ is cleared from brain interstitial fluid (ISF) and cerebrospinal fluid (CSF) by LRP2-mediated transcytosis across epithelial and endothelial barriers. At physiological ISF/CSF levels, apoJ is rapidly transported by LRP2 across the blood-brain barrier (BBB). Importantly, apoJ also substantially enhances clearance from the brain of amyloidogenic Aβ isoforms (i.e., higher β-sheet content such as Aβ42) as apoJ-Aβ by LRP2-mediated transport.06-18-2009
20090156523METHODS AND COMPOSITIONS FOR MODULATING FOXO1 ACTIVITY AND INSULIN SIGNALING - The invention provides methods for modulating insulin signaling pathway and methods for treating insulin resistance. The methods employ agents which modulate FOXO1 subcellular localization. The invention also provides methods of screening for compounds that modulate insulin signaling related activities such as gluconeogenesis and cell proliferation.06-18-2009
20090156522Pancreatic Cancer Genes - The present invention provides the art with the DNA coding sequences of polynucleotides that are up-or-down-regulated in cancer and dysplasia. These polynucleotides and encoded proteins or polypeptides can be used in the diagnosis or identification of cancer and dysplasia. Inhibitors of the up-regulated polynucleotides and proteins can decrease the abnormality of cancer and dysplasia. Enhancing the expression of down-regulated polynucleotides or introducing down-regulated proteins to cells can decrease the growth and/or abnormal characteristics of cancer and dysplasia.06-18-2009
20090156521Gpr17 modulators,method of screening and uses thereof - The invention provides GPR17 modulators, methods of screening and use thereof for diagnosis and therapy of diseases or dysfunctions involving GPR17 activation, particularly ischemic brain damage.06-18-2009
20090156520Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA - Introduction of double stranded RNA into cells, cell culture, organs and tissues, and whole organisms, particularly vertebrates, specifically attenuates gene expression.06-18-2009
20090156519Modified KSA and Uses Thereof - The present invention relates to a nucleic acid encoding a polypeptide and the use of the nucleic acid or polypeptide in preventing and/or treating cancer. In particular, the invention relates to improved vectors for the insertion and expression of foreign genes encoding tumor antigens for use in immunotherapeutic treatment of cancer.06-18-2009
200802075542-5A Analogs and their Methods of Use - This invention relates to the fields of organic chemistry, pharmaceutical chemistry, biochemistry, molecular biology and medicine. In particular it relates to compounds that activate RNaseL, and to the use of the compounds for treating and/or ameliorating a disease or a condition, such as a viral infection.08-28-2008
20090192100NOVEL USE OF SPIEGELMERS - The present invention relates to the use of a L-nucleic acid as intracellularly active agent.07-30-2009
20090137507RNA INTERFERENCE MEDIATED INHIBITION OF ANGIOPOIETIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating Angiopoietin gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of Angiopoietin gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of Angiopoietin genes, such as Angiopoietin-1 (Ang-1), Angiopoietin-2 (Ang-2), Angiopoietin-3 (Ang-3), and Angiopoietin-4 (Ang-4).05-28-2009
20090124564Compositions and methods for regulating apoptosis - Methods and pharmaceutical compositions for regulating apoptosis and treating pathologies associated with disregulated apoptosis are provided. Specifically the present invention provides agents capable of modulating the expression of an MCD-containing protein (e.g., Mtch2) capable of tBID binding activity and/or the tBID binding activity of the MCD-containing protein.05-14-2009
20090124569INHIBITION AND TREATMENT OF PROSTATE CANCER METASTASIS - The present invention provides compounds and methods of inhibiting and treating metastatic prostate cancer. The compounds include MEK4 inhibitors. In another aspect the invention provides methods of identifying inhibitors of metastatic prostate cancer by screening for inhibitors of MEK4.05-14-2009
20090124568USE OF STEM CELLS TO GENERATE INNER EAR CELLS - This invention relates generally to methods and compositions for inducing stem cell or progenitor cell differentiation, and more particularly to methods and compositions for inducing differentiation of stem cells and/or progenitor cells into cells that function within the inner ear.05-14-2009
20090124566METHODS AND COMPOSITIONS FOR THE TREATMENT OF ERYTHROCYTE DISEASES - Methods to determine the susceptibility of a subject to erythrocyte diseases are provided. The methods comprise determining the miRNA compositions of erythrocytes from the subject. The present invention has discovered that erythrocytes comprise microRNA (miRNA) populations and the populations can be profiled or analyzed and used to determine the susceptibility for disease. The miRNA compositions can also be used to determine the severity of erythrocyte disease, and the features and clinical phenotypes of the erythrocyte disorders. Also provided are pharmaceutical compositions comprising erythrocyte miRNAs and methods for the treatment of a subject with an erythrocyte disease. In other embodiments of the invention, the miRNAs can be used to increase the life-span of erythrocytes through the introduction of an erythrocyte miRNA.05-14-2009
20090124565COMPOSITION COMPRISING AT LEAST ONE NUCLEOSIDIC MOIETY AS A THERAPEUTIC AGENT, AND CKC - Composition comprising at least one nucleosidic moiety and cetalkonium chloride and pharmaceutical use thereof for prevention, treatment or relief of eye, lung, and/or respiratory tract conditions.05-14-2009
20080312179High level expression of recombinant human erythropoietin having a modified 5'-UTR - The present invention provides an expression construct capable of producing high levels of erythropoietin in mammalian cells. More particularly, the expression construct includes an erythropoietin coding region fused to a unique 5′-UTR sequence and a truncated 3′-UTR. The present invention also provides methods of synthesizing large amounts of erythropoietin and in increasing serum erythropoietin level in individuals in need thereof.12-18-2008
20080227735Aptamers Selected From Live Tumor Cells and the Use Thereof - The invention relates to aptamers selected from live tumor cells and to the use thereof for diagnosis and treatment of certain cancers and other pathologies.09-18-2008
20090005331AChE antisense oligonucleotide as an anti-inflammatory agent - Disclosed is a novel use for AChE antisense oligonucleotides as anti-inflammatory agents, wherein said oligonucleotides are preferably as denoted by SEQ ID NO:1, SEQ ID NO:2 and SEQ ID NO:7. Methods of treatment of inflammatory conditions, as well as fever, and particularly inflammation-associated neuropathies such as Guillain-Barré Syndrome, are described.01-01-2009
20090042828METHODS AND COMPOSITIONS FOR TREATING GAIN-OF-FUNCTION DISORDERS USING RNA INTERFERENCE - The present invention relates to novel methods for treating dominant gain-of-function diseases. The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS), with RNAi agent. The invention further provides RNAi resistant replacement genes containing mismatches with their respective RNAi agents. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention.02-12-2009
20090176727DOUBLE-STRANDED RNA STRUCTURES AND CONSTRUCTS, AND METHODS FOR GENERATING AND USING THE SAME - The present invention relates to novel double-stranded RNA (dsRNA) structures and dsRNA expression constructs, methods for generating them, and methods of utilizing them for silencing genes. Desirably, these methods specifically inhibit the expression of one or more target genes in a cell or animal (e.g., a mammal such as a human) without inducing toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, such as the function of a cell, the expression of a gene, or the biological activity of a target polypeptide.07-09-2009
20090176722Androgen-regulated PMEPA1 gene and polypeptides - This invention relates to the androgen-regulated gene, PMEPA1, and proteins encoded by this gene, including variants and analogs thereof. Also provided are other androgen-regulated nucleic acids, a polynucleotide array containing these androgen-regulated nucleic acids, and methods of using the polynucleotide array in the diagnosis and prognosis of prostate cancer. 07-09-2009
20090306005COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF PCSK9 - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.12-10-2009
20090118218Biocompatible Hydrogels - A biocompatible macromer composition is provided which includes a first polymer possessing a first nucleoside, and a second polymer possessing a second nucleoside that is complementary to the first nucleoside and capable of undergoing base pairing with the first nucleoside. The biocompatible macromer composition can be used as an adhesive or sealant in human and/or animal medical applications.05-07-2009
20090118217Genetic polymorphisms associated with myocardial infarction, methods of detection and uses thereof - The present invention is based on the discovery of genetic polymorphisms that are associated with myocardial infarction. In particular, the present invention relates to nucleic acid molecules containing the polymorphisms, variant proteins encoded by such nucleic acid molecules, reagents for detecting the polymorphic nucleic acid molecules and proteins, and methods of using the nucleic acid and proteins as well as methods of using reagents for their detection.05-07-2009
20090118216ANAPLASTIC LYMPHOMA KINASE (ALK) AS ONCOANTIGEN FOR LYMPHOMA VACCINATION - Use of intracytoplasmatic domain of Anaplastic Lymphoma Kinase (ALK) protein and/or a nucleic acid molecule encoding for the intracytoplasmatic domain of Anaplastic Lymphoma Kinase (ALK) protein, of any species, for the preparation of a medicament, preferably a vaccine, for the treatment and/or prevention of a tumor in a subject, preferably a lymphoma.05-07-2009
20080318883Anti-aging supplement - An anti-aging supplement is disclosed having effective amounts of β1,3D glucan, deoxyribonucleic acid, and body's hyaluronan-yielding row material composed of at least one amino sugar selected from a group consisting of N-acetylglucosamine and glucosamine salts.12-25-2008
20090093437RNA INTERFERENCE MEDIATED INHIBITION OF CHECKPOINT KINASE-1 (CHK-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating checkpoint kinase (e.g., checkpoint kinase-1 or CHK-1) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of checkpoint kinase gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of checkpoint kinase genes.04-09-2009
20090093436RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR CELL ADHESION MOLECULE (VCAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating vascular cell adhesion molecule gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of vascular cell adhesion molecule gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of vascular cell adhesion molecule genes, such as vascular cell adhesion molecule-1 (VCAM-1).04-09-2009
20090093431RNA INTERFERENCE MEDIATED INHIBITION OF NOGO AND NOGO RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This inventon relates to compounds, compositions, and methods useful for modulating NOGO and/or NOGO receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of NOGO and/or NOGO receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of NOGO and/or NOGO receptor genes, such as NOGO-A, NOGO-B, NOGO-C, NOGO-66 receptor, NI-35, NI-220, NI-250, myelin-associated glycoprotein, tenascin-R, and NG-204-09-2009
20090093429COMBINATION THERAPY FOR TREATING CANCER - The invention relates to treatment of primary and secondary tumors using a therapeutic combination including ionizing radiation and gene therapy comprising mutant-LIGHT. Methods of treating a tumor and inducing an anti-tumor immune response are also provided.04-09-2009
20090093426RNAI MODULATION OF SCAP AND THERAPEUTIC USES THEREOF - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a SCAP gene (Human SCAP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a SCAP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition; and methods for inhibiting the expression of a SCAP gene in a cell.04-09-2009
20080287384Methods of Identifying Agents Having Antiangiogenic Activity - Provided are assays and methods of identifying antiangiogenic agents including contacting an endothelial cell with a putative antiangiogenic agent and assaying for activation of Rap-1 in the endothelial cell. Also provided are methods of inhibiting angiogenesis and treating conditions associated with improper angiogenesis. Compositions comprising activators of Rap-1 and methods of activating the Rap-1 signaling pathway are also provided. Methods of inhibiting chemotaxis and angiogenesis by contacting cells with Rho inhibitors are provided.11-20-2008
20090069261GENE THERAPY FOR SPINAL CORD DISORDERS - This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.03-12-2009
20090069256Enhancing protein expression - Modified polynucleotide compositions providing enhanced gene expression and methods for preparing said compositions are disclosed. Methods of using the compositions, such as in screening assays, diagnostic tools, kits, etc. and for prevention and/or treatment of diseases and disorders are also disclosed.03-12-2009
20090029930Methods of gene therapy using nucleic acid sequences for ATP-binding cassette transporter - The present invention provides nucleic acid and amino acid sequences of an ATP binding cassette transporter and mutated sequences thereof associated with macular degeneration. Methods of detecting agents that modify ATP-binding cassette transporter comprising combining purified ATP binding cassette transporter and at least one agent suspected of modifying the ATP binding cassette transporter an observing a change in at least one characteristic associated with ATP binding cassette transporter. Methods of detecting macular degeneration is also embodied by the present invention.01-29-2009
20090163438GANKYRIN - Gankyrin having the amino acid sequence as set forth in SEQ ID NO: 2, or modified gankyrin comprising an amino acid sequence modified by the deletion and/or addition of one or a plurality of amino acids and/or the substitution with other amino acids in the amino acid sequence of SEQ ID NO: 2 and retaining the biological activity of gankyrin, a gene encoding it, and a method of preparing said protein and uses thereof.06-25-2009
20090023672AGE-2 APTAMER - An AGE-2 aptamer which binds to a glyceraldehyde-derived advanced glycation end product (AGE-2) but not to human serum albumin and comprises at least 35 bases and in which the cytosine content in the bases is at least 35%, or the guanine content in the bases is at least 32%. Since the AGE-2 aptamer can be used for detecting AGE-2, it can be used as a reagent for detection/diagnosis, and an agent for prevention/treatment of AGE-2 involved diseases such as: diabetic complications such as diabetic retinopathy, diabetic nephropathy, and diabetic neuropathy; neurodegenerative diseases such as Alzheimer's disease; and proliferation, metastasis, and invasion of malignant tumors.01-22-2009
20090012033Delivery of Biologically Active Materials Using Core-Shell Tecto(Dendritic Polymers) - The present invention concerns core-shell tecto (dendritic polymers) that are associated with biologically active materials (such as nucleic acids for use for RNAi and in transfection). Also included are formulations for their use. The constructs are useful for the delivery of drugs to an animal or plant and may be in vivo, in vitro or ex vivo.01-08-2009
20090012030RNAi-MEDIATED INHIBITIN OF HTRA1 FOR TREATMENT OF MACULAR DEGENERATION - RNA interference is provided for inhibition of HTRA1 mRNA expression for treating patients with an HTRA1-mediated ocular disorder. In particular, methods are provided for treating age-related macular degeneration (AMD) and using interfering RNA molecules that attenuate expression of HTRA1 in patients having AMD or at risk of developing AMD.01-08-2009
20090012028Polyglutamic acids functionalized by cationic groups and hydrophobic groups and applications thereof, in particular therapeutic applications thereof - The present invention relates to novel biodegradable materials based on modified polyamino acids that are useful in particular in the vectorization of active principle(s) (APs). The invention is also directed to novel pharmaceutical, cosmetic, health-food or plant-protection compositions based on these polyamino acids.01-08-2009
20090012027BIODEGRADABLE POLYPHOSPHORAMIDATES FOR CONTROLLED RELEASE OF BIOACTIVE SUBSTANCES - The present invention is directed to a series of new polycationic biodegradable polyphosphoramidates. Process for making the polymers, compositions containing these polymers and bioactive ligands to enhance the cellular uptake ad intracellular trafficking, articles and methods for delivery of drugs and genes using these polymers are described. A gene delivery system based on these polymers is prepared by complex coacervation of nucleic acid (DNA or RNA) with polymers. Targeting ligands and molecules that could facilitate gene transfer can be conjugated to polymers to achieve selective and enhanced gene delivery. The current invention also provides a complex composition with buffering capacity.01-08-2009
20090012032GENES AND POLYPEPTIDES RELATING TO HUMAN COLON CANCERS - The present application provides novel human genes RNF43 whose expression is markedly elevated in colorectal cancers, as well as CXADRL1 and GCUD1 whose expression is markedly elevated in gastric cancers compared to corresponding non-cancerous tissues. The genes and polypeptides encoded by the genes can be used, for example, in the diagnosis of a cell proliferative disease, and as target molecules for developing drugs against the disease.01-08-2009
20090012026Association Between the Tdoa Gene and Osteoarthritis - The present invention arises from the identification of an association between the TDOA gene and osteoarthritis (OA). It therefore relates to diagnostic techniques for determining a patient's susceptibility to develop OA by detecting all or part of the TDOA gene, its precursors or products (mRNA, cDNA, genomic DNA, or protein). In particular, the invention relates to methods and materials for analysing allelic variation in the TDOA gene, and to the use of TDOA polymorphisms in the identification of an individuals' risk to develop OA. The TDOA protein has been found to bind to α-paxillin, a protein involved in integrin signal transduction which itself is a process with strong links to OA. The invention is thus also directed to methods for identifying modulators of OA, which modulate the TDOA gene or interfere with TDOA:α-paxillin binding.01-08-2009
20090012025Methods and Compositions for Treatment of Sepsis - Methods of treatment of sepsis are disclosed. These methods comprise administering to a subject a composition comprising at least one siRNA directed against at least one gene encoding a pro-apoptotic polypeptide. The pro-apoptotic polypeptide, in some aspects, can be other than Fas or caspase-8. In some embodiments, an siRNA can be directed against a pro-apoptotic component of the mitochondrial pathway, such as a pro-apoptotic bcl-2 protein. In some aspects, an siRNA can be directed against a BH3-only bcl-2 protein, while in other aspects, siRNAs can be directed against multi BH domain Bcl-2 family members such as bax and bak. In some embodiments, an siRNA can be directed against a death receptor pathway molecule such as FADD. In various configurations, a composition can also comprise a cationic lipid such as DOTAP, or nanoparticles comprising a cyclodextrin-containing polycation and a polymer such as a poly(ethylene glycol).01-08-2009
20090012024Stem Cell Markers - We disclose gene markers of stem cells, typically prostate stem cells, and in particular cancer stem cells, for example prostate cancer stem cells; therapeutic agents and diagnostic assays based on said stem cell genes; and including screening assays to identify therapeutic agents.01-08-2009
20090012022Hybrid Interfering Rna - The invention relates to a hybrid interfering RNA molecule comprising a duplex RNA and a single stranded DNA molecule and its use in the ablation of mRNA and in polymerase chain reactions.01-08-2009
20090012020Inhibiting formation of atherosclerotic lesions - The invention features a method of inhibiting formation of atherosclerotic lesions by administering to a mammal, e.g., a human patient who has been identified as suffering from or at risk of developing atherosclerosis, a compound that reduces expression or activity of AFABP.01-08-2009
20090012018Inhibition of pancretic cancer cell growth - The present invention relates to compositions and methods for inhibition of signal transduction pathways in cancer cells. In particular, the present invention provides methods and compositions comprising Wnt and Hedgehog pathway inhibitors for reducing proliferation of adenocarcinoma cells.01-08-2009
20090012017Polypeptide Complex that Regulates Cell Cycle and Anergy - An active ubiquitin E3 ligase, GRAIL, is crucial in the induction of anergy in cells of the immune system, and in the regulation of cellular proliferation. GRAIL is shown to associate with, and be regulated by Otubain isoforms, including OTUBAIN-1 (DOG, the Destabilizer of GRAIL) and an alternative reading frame splice variant of OTUBAIN-1 (SOG, the Stabilizer of GRAIL). These proteins play opposing roles in the regulation of GRAIL auto-ubiquitination and consequently on its ability to induce anergy and regulate cellular proliferation. DOG serves as an adaptor protein, recruiting the DUB USP8. One major substrate for USP8 is the Ras exchange factor Ras-GRF1, and this protein can be found in a complex with USP8 and GRAIL, which complex is ubiquitinated by GRAIL.01-08-2009
20090012016Short Interfering Rna and Micro-Rna Compounds and Methods of Designing, Making, and Using the Same - Methods of identifying, designing and synthesizing uniquely targeting siRNA nucleotide sequences for a target mRNA sequence of a target species are disclosed. Methods of identifying, designing and synthesizing miRNA nucleotide sequences that does not function as a siRNA nucleotide sequence for mRNA of a target species are disclosed. Method of inhibiting expression of a target mRNA molecule are disclosed sequence. siRNA molecules including uniquely targeting siRNA molecules and RISCs comprising the same are disclosed. miRNA molecules that do not function as siRNA nucleotide sequences for mRNA of a target species are disclosed.01-08-2009
20080280844METHODS AND COMPOSITIONS FOR THE DIAGNOSIS AND TREATMENT OF EWING'S SARCOMA - The present invention relates to methods and compositions for the detection and treatment of Ewing's sarcoma. In particular, the methods of detection relate to measuring in Ewing's sarcoma cells the expression of the NKX2.2 gene, as well as targets genes downstream of NKX2.2. The compositions and method of treatment for Ewing's sarcoma involve therapeutic agents that target the expression of the NKX2.2 gene or block the activity of the NKX2.2 protein. Also provided are methods of screening therapeutic agents that affect the expression of the NKX2.2 gene.11-13-2008
20080227742Photocleavable oligonucleotide and uses thereof - This invention relates to methods and compositions of oligonucleotide constructs having a photocleavable linker. Specifically, provided herein are methods and compositions utilizing a photocleavable linker, which when exposed to light modulates the expression of genes.09-18-2008
20080269157Prostate cancer-specific alterations in ERG gene expression and detection and treatment methods based on those alterations - Alterations in ERG gene expression can be observed in patients with prostate cancer. Specific ERG isoforms are associated with, or involved in, prostate cancer. Compositions comprising these isoforms provide therapeutic benefit and can be used in methods of detecting, diagnosing, prognosing, and treating prostate cancer. These compositions provide biomarkers for detecting the expression of combinations of the PSA/KLK3, PMEPA1, NKX3.1, ODC1, AMD1, and ERG genes.10-30-2008
20090042824Methods and Kits for Linking Polymorphic Sequences to Expanded Repeat Mutations - Methods and kits are provided for determining which single nucleotide polymorphism (“SNP”) variant of an allele of a heterozygous patient is on the same allele as a disease-causing mutation that is at a remote region of the gene's mRNA comprising a) an allele specific reverse transcription reaction using an allele specific primer which recognizes one SNP variant, wherein further the 3′ end of the primer is positioned at the SNP nucleotide position, and b) analysis of the resulting cDNA product from the reverse transcription reaction at the region of the mutation to determine the presence or absence of the mutation on this allele specific cDNA product, wherein the allele specific primer is shorter than about 20 nucleotides.02-12-2009
20090192112COMPOSITIONS AND METHODS FOR TREATING CANCER - The present invention relates to therapeutic targets for cancer. In particular, the present invention relates to small molecules and nucleic acids that target ATDC (TRIM29) expression in cancer with ATDC overexpression.07-30-2009
20080293663CELLULAR GENES REGULATED BY HIV-1 INFECTION AND METHODS OF USE THEREOF - This invention provides cellular gene products which have anti-apoptotic activity in HIV-1 infected cells and provides agents for the inhibition of the cellular gene products.11-27-2008
20080293662RNAi MODULATION OF THE BCR-ABL FUSION GENE AND USES THEREOF - The invention relates to compositions and methods for modulating the expression of Bcr-Abl, and more particularly to the down-regulation of Bcr-Abl mRNA and Bcr-Abl protein levels by oligonucleotides via RNA interference, e.g., chemically modified oligonucleotides.11-27-2008
20080293661METHOD FOR TREATING CHRONIC LYMPHOID LEUKEMIA - Embodiments of the invention relates to the treatment of chronic lymphoid leukemia. The invention provides treatment, diagnostic, and drug discovery strategies for chronic lymphoid leukemia. Reconstitution of RhoH expression in vitro inhibits neoplastic proliferation and the process of trans-endothelial migration that underlies much of the pathology of CCL. RhoH reconstitution also limits malignant progression in vivo. Therefore, RhoH reconstitution represents a new therapeutic strategy in the fight against chronic lymphoid leukemia.11-27-2008
20080293660CHEMICALLY-DEFINED NON-POLYMERIC VALENCY PLATFORM MOLECULES AND CONJUGATES THEREOF - Chemically-defined, non-polymeric valency platform molecules and conjugates comprising chemically-defined valency platform molecules and biological or chemical molecules including polynucleotide duplexes of at least 20 base pairs that have significant binding activity for human lupus anti-dsDNA autoantibodies.11-27-2008
20080300207PROTEIN PRODUCTION - The invention concerns the field of protein production and cell culture technology. CERT is identified as a novel in vivo PKD substrate. Phosphorylation on serine 132 by PKD decreases the affinity of CERT towards its lipid target phosphatidylinositol 4-phosphate at Golgi membranes and reduces ceramide transfer activity, identifying PKD as a regulator of lipid homeostasis. The present invention shows that CERT in turn is critical for PKD activation and PKD dependent protein cargo transport to the plasma membrane. The interdependence of PKD and CERT is thus a key to the maintenance of Golgi membrane integrity and secretory transport.12-04-2008
20090012019Methods and Compositions Related to TR4 - Disclosed are compositions and methods related to TR4 and aging.01-08-2009
20090131352Aptamers comprising arabinose modified nucleotides - Nucleic acid ligands (or aptamers) that form a G-tetrad containing at least one arabinose modified nucleotide are provided. Preferably, the arabinose modified nucleotide is 2′-deoxy-2′-fluoroarabinonucleotide (FANA) nucleotide. Methods of using aptamers the aptamers of the claimed invention are also provided.05-21-2009
20090075924MODULATION OF APOLIPOPROTEIN (a) EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of apolipoprotein(a). The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein(a). Methods of using these compounds for modulation of apolipoprotein(a) expression and for diagnosis and treatment of disease associated with expression of apolipoprotein(a) are provided.03-19-2009
20090012023DECOY-OLIGNUCLEOTIDE INHIBITION OF CD40-EXPRESSION - The present invention relates to decoy oligonucleotides with the nucleic acid sequence according to SEQ ID NO: 1 to 36 and their use as pharmaceutical agents.01-08-2009
20090156534GLOBIN LENTIVIRAL VECTORS FOR TREATMENT OF DISEASE - The invention provides compositions and methods for the treatment or prevention of disease, including, for example, β-thalassemia, anemias (e.g., sickle cell anemia) and other hemoglobinopathologies.06-18-2009
20090156527ANTIVIRAL OLIGONUCLEOTIDES TARGETING VIRAL FAMILIES - Random sequence oligonucleotides that have antiviral activity are described, along with their use as antiviral agents. In many cases, the oligonucleotides are greater than 40 nucleotides in length. Also described are methods for the prophylaxis or treatment of a viral infection in a human or animal, and a method for the prophylaxis treatment of cancer caused by oncoviruses in a human or animal. The methods typically involve administering to a human or animal in need of such treatment, a pharmacologically acceptable, therapeutically effective amount of at least oligonucleotide that does not act by a sequence complementary mode of action.06-18-2009
20080300206Alpha-Synuclein Kinase - The invention provides agents and methods for treatment of diseases associated with Lewy body diseases (LBDs) in the brain of a patient. Preferred agents include inhibitors of PLK2 kinase.12-04-2008
20090186844SiRNA DELIVERY INTO MAMMALIAN NERVE CELLS - The present invention relates to methods of affecting expression of a target gene, suitably brain-derived neurotrophic factor (BDNF) or related genes in a nerve cell in the central nervous system of a mammal. The method includes formulating and delivering an siRNA composition to a target site on the mammal to affect expression of the target gene in the nerve cell, wherein the target site is cerebrospinal space or muscle tissue innervated by a nerve cell, to down-regulate the target gene. Also disclosed are kits for use in practicing the novel methods of in vivo siRNA.delivery into target cells and gene regulation.07-23-2009
20090149403siRNA silencing of genes expressed in cancer - The present invention provides nucleic acid-lipid particles comprising siRNA molecules that silence genes expressed in cancer (e.g., Eg5, EGFR or XIAP) and methods of using such nucleic acid-lipid particles to silence Eg5, EGFR or XIAP gene expression.06-11-2009
20080312174WATER SOLUBLE CROSSLINKED POLYMERS - Compositions for siRNA delivery are described which include water soluble degradable crosslinked cationic polymers having a water soluble polyethylene glycol component, a cationic polyethyleneimine component and a degradable unit component. The composition may be used to deliver siRNA to cells, particularly cancer cells. The composition may be applied to a solid surface such as a multiwell plate so that the delivery of siRNA may be carried out on the solid surface.12-18-2008
20080306014Agents, compositions and methods for treating pathologies in which regulating an ache-associated biological pathway is beneficial - The present invention provides agents which are capable of regulating the function of a micro-RNA component which can be used to regulate an AChE-associated biological pathway. In addition, the present invention provides methods and pharmaceutical compositions for the treatment of various pathologies related to AChE-associated biological pathways such as apoptosis, aberrant cholinergic signaling, abnormal hematopoietic proliferation and/or differentiation, cellular stress, exposure to inflammatory response-inducing agents, and/or exposure to organophosphates or other AChE inhibitors.12-11-2008
20090036395RNA INTERFERENCE SUPPRESSION OF NEURODEGENERATIVE DISEASES AND METHODS OF USE THEREOF - The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence that encodes poly-glutamine repeat diseases, and methods of using these RNAi molecules.02-05-2009
20090137512RNA Interference Mediated Inhibition of Cyclin D1 Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating cyclin (e.g., cyclin D1) and/or cyclin dependent kinase (CDK) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of cyclin (e.g., cyclin D1) and/or cyclin dependent kinase (CDK) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of cyclin (e.g., cyclin D1) and/or cyclin dependent kinase (CDK) genes.05-28-2009
20090048195COMPOSITIONS AND METHODS OF SPHINGOSINE KINASE INHIBITORS FOR USE THEREOF IN CANCER THERAPY - The present invention relates to Sphingosine kinase inhibitors that are useful for treating various cancers. The invention specifically relates to compositions and methods of SPK inhibitors, including siRNAs, which specifically block gene expression of SPK and induce apoptosis in a variety of cancers.02-19-2009
20080207551MODIFIED OLIGONUCLEOTIDES CONTAINING DIPHOSPHODIESTER INTERNUCLEOTIDE LINKAGES - The synthesis and biochemical utility of modified oligonucleotides containing diphosphodiester internucleotide linkages. The synthesis of these compounds was carried out using diphosphitylating reagents. Oligonucleotides containing diphosphate diester bridges wherein said oligonucleotides are synthesized via a solid-phase synthesis strategy to form modified oligonucleotides. Diphosphitylating, triphosphitylating, tetraphosphitylating, β-triphosphitylating, bifunctional diphosphitylating, bifunctional triphosphitylating, and bifunctional tetraphosphitylating reagents wherein, the phosphorus atoms are linked together through oxygen, sulfur, amino, or methylene groups and/or are substituted with chlorine, diisopropylamine and cyanoethoxy groups.08-28-2008
20090048197Lipid Nanoparticle Based Compositions and Methods for the Delivery of Biologically Active Molecules - The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), short hairpin RNA (shRNA), and RNAi inhibitor molecules, to relevant cells and/or tissues, such as in a subject or organism. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).02-19-2009
20080214484NUCLEIC ACIDS ENCODING SARCOCYSTIS NEURONA ANTIGEN AND USES THEREOF - The present invention provides novel isolated nucleic acids encoding antigenic proteins derived from 09-04-2008
20090156539ANTISENSE OLIGONUCLEOTIDES (ODN) AGAINST SMAD7 AND USES THEREOF IN MEDICAL FIELD - The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.06-18-2009
20090005330Methods and Compositions to Inhibit P2x7 Receptor Expression - Methods and compositions for the downregulation of P2X7 receptor expression or activity are disclosed. Preferred compositions comprise siNA. The methods and compositions are useful in the treatment of diseases characterised by increased 112X7 receptor activity, such as neuronal degeneration, Alzheimer's disease, inflammatory diseases, and some cancers.01-01-2009
20090099122USE OF CpG OLIGODEOXYNUCLEOTIDES TO INDUCE EPITHELIAL CELL GROWTH - This disclosure provides a method of inducing epithelial cell growth. The method includes administering an effective amount of a K-type CpG oligonucleotide, thereby inducing epithelial cell growth. The epithelial cell can be in vivo or in vitro. Methods are also provided for inducing wound healing in a subject. The methods include administering to the subject a therapeutically effective amount of at least on K-type CpG ODN.04-16-2009
20090099125PAN CANCER ONCOLYTIC VECTORS AND METHODS OF USE THEREOF - Replication-competent adenoviral vectors which selectively replicate in cancer cells are provided. The replication-competent viral vectors comprise an E2F responsive promoter and/or a telomerase promoter operatively linked to an adenoviral coding region. The replication-competent adenoviral vectors effectively replicate in a variety of types of cancer cells and find broad utility in the treatment of cancer.04-16-2009
20090099111Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.04-16-2009
20090099115RNA INTERFERENCE MEDIATED INHIBITION OF MYC AND/OR MYB GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating Myc and/or Myb gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of Myc and/or Myb gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of Myc and/or Myb (e.g., c-Myc, N-Myc, L-Myc, c-Myb, a-Myb, b-Myb, and v-Myb) genes. The small nucleic acid molecules are useful in the treatment of cancer and other diseases and disorders.04-16-2009
20090099121RNA INTERFERENCE MEDIATED INHIBITION OF MATRIX METALLOPROTEINASE 13 (MMP13) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating matrix metalloproteinase (e.g., MMP13) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of MMP13 genes.04-16-2009
20090099120RNA INTERFERENCE MEDIATED INHIBITION OF MUSCARINIC COLINERGIC RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention relates to compounds, compositions, and methods useful for modulating the expression of genes associated with respiratory and pulmonary disease, such as cholinergic muscarinic receptor genes, using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of cholinergic muscarinic receptor genes, or other genes involved in pathways of cholinergic muscarinic receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of M3 muscarinic acetylcholine receptor or cholinergic receptor muscarinic 3 (CHRM3).04-16-2009
20090099116RNA INTERFERENCE MEDIATED INHIBITION OF FOS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating c-Fos gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of c-Fos gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of c-Fos genes. The small nucleic acid molecules are useful in the treatment of cancer, proliferative diseases or conditions, inflammatory diseases or conditions, allergic diseases or conditions, infectious diseases or conditions, autoimmune diseases or conditions, or transplantation/allograft rejection in a subject or organism.04-16-2009
20090099113Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.04-16-2009
20090099109INTERFERING RNAS AGAINST THE PROMOTER REGION OF P53 - The present invention relates to the inhibition of p53 transcription by interfering with the activity of a p53 promoter using inhibitory double-stranded RNAs. Use of these inhibitory RNAs in the treatment of cancers also is disclosed.04-16-2009
20090099107Methods and Compositions for Inhibition of Nuclear Factor kappaB - A series of p105-based NF-κB super repressors, designated p-105(sr), have been designed. The p105(sr), no longer generates p50 and undergoes signal-induced degradation, effectively inhibiting all NF-κB activities. Additionally, p105(sr) significantly enhances tumor necrosis factor alpha (TNF-α)-mediated killing of MT1/2 skin papilloma cells when p50 homodimer activity is elevated. p105(sr) is an effective NF-κB super repressor with a broader range than other currently available IkBα super repressors. The novel repressor can be used in cells where a noncanical NF-κB activity is dominant or multiple NF-κB activities are activated.04-16-2009
20090137510RNA INTERFERENCE MEDIATED INHIBITION OF NF-KAPPA B/ REL-A GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating NF-kappa B, REL-A, REL-B, REL, NKkappaB1, or NFkappaB2 gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of NF-kappa B, REL-A, REL-B, REL, NKkappaB1, or NFkappaB2 gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of NF-kappa B, REL-A, REL-B, REL, NKkappaB1, or NFkappaB2 genes, such as NF-kappa B and/or REL-A.05-28-2009
20090192116COMPOSITIONS AND METHODS FOR THE SUPPRESSION OF TARGET POLYNUCLEOTIDES FROM THE FAMILY APHIDIDAE - Methods and compositions are provided which employ a silencing element that, when ingested by a pest, such as a pest from the Aphididae family, they are capable of decreasing the expression of a target sequence in the pest. In specific embodiments, the decrease in expression of the target sequence controls the pest and thereby the methods and compositions are capable of limiting damage to a plant. The present invention provides target polynucleotides for specific protein classes and also target polynucleotides as set forth in SEQ ID NOS:1, 2, 3, 4, 5, 6, 7, 8, 9, 10 or active variants or fragments thereof, wherein a decrease in expression of one or more the sequences in the target pest controls the pest (i.e., has insecticidal activity). Further provided are silencing elements which when ingested by the pest decrease the level of the target polypeptide and thereby control the pest. In specific embodiment, the pest is 07-30-2009
20090082292ANTISENSE OLIGONUCLEOTIDES CAPABLE OF INHIBITING THE FORMATION OF CAPILLARY TUBES BY ENDOTHELIAL CELLS AND METHODS OF TREATING OPHTHALMIC AND DERMATOLOGICAL DISEASES - A pharmaceutical composition blocks angiogenesis and contains as an active agent at least one nucleotide sequence from nucleic acid molecule SEQ ID NO. 3, fragments thereof containing at least twelve contiguous nucleotides and derivatives thereof; and nucleic acid sequences containing at least twelve contiguous nucleotides of the nucleic acid molecule SEQ ID NO 30 and derivatives thereof.03-26-2009
20090137514METHODS AND COMPOSITIONS FOR SILENCING GENES WITHOUT INDUCING TOXICITY - The present invention provides methods of post-transcriptional gene silencing which involve the use of a first dsRNA having substantial sequence identity to a target nucleic acid and a short, second dsRNA which inhibits dsRNA-mediated toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, including the function of a cell, the expression of a gene, or the biological activity of a target polypeptide.05-28-2009
20080269152Nucleic acid binding oligonucleotides - The present application pertains to products and methods related to the ability of short nucleotide oligomers to bind the tertiary or globular structure of nucleic acids. This application discloses libraries of short oligomers and methods for using these libraries.10-30-2008
20090149411IMMUNOSTIMULATORY OLIGONUCLEOTIDES WITH MODIFIED BASES AND METHODS OF USE THEREOF - Immunomodulatory oligonucleotide compositions are disclosed. These oligonucleotides comprise an immunostimulatory hexanucleotide sequence comprising a modified cytosine. These oligonucleotides can be administered in conjunction with an immunomodulatory peptide or antigen. Methods of modulating an immune response upon administration of the oligonucleotide comprising a modified immunostimulatory sequence are also disclosed.06-11-2009
20090149410Methods, compositions, and kits relating to chitinases and chitinase-like molecules and inflammatory disease - The present invention includes compositions and methods for the treatment of inflammatory disease (e.g. asthma, COPD, inflammatory bowel disease, atopic dermatitis, atopy, allergy, allergic rhinitis, scleroderma, and the like), relating to inhibiting a chitinase-like molecule. The invention further includes methods to identify new compounds for the treatment of inflammatory disease, including, but not limited to, asthma, COPD and the like. This is because the present invention demonstrates, for the first time, that expression of IL-13, and of a chitinase-like molecule, mediates and/or is associated with inflammatory disease and that inhibiting the chitinase-like molecule treats and even prevents, the disease. Thus, the invention relates to the novel discovery that inhibiting a chitinase-like molecule treats and prevents an inflammatory disease.06-11-2009
20090149409Tetracycline-Regulated Adeno-Associated Viral (AAV) Vectors for Gene Delivery to the Nervous System - A vector and a method are provided for delivering a nucleic acid to a nervous system cell. The vector includes a first nucleic acid, a second nucleic acid, inverted terminal repeats of adeno-associated virus, and a tetracycline-off regulatable promoter system that includes a first promoter operably linked to the first nucleic acid and a second promoter operably linked to the second nucleic acid. The promoters drive expression in opposite directions and away from the inverted terminal repeats. The method includes providing a recombinant adeno-associated viral (rAAV) vector and administering the vector to a nervous system cell. Expression of a product from the first nucleic acid is regulatable by the promoter system.06-11-2009
20090149408RNA INTERFERENCE MEDIATED INHIBITION OF TELOMERASE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating telomerase gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of telomerase gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of telomerase genes, such as telomerase template RNA (TERC/TR), or a telomerase protein (TERT).06-11-2009
20090149407RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER'S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention concerns methods and reagents useful in modulating BACE gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against beta-secretase (BACE), amyloid precursor protein (APP), pin-1, presenillin 1 (PS-1) and/or presenillin 2 (PS-2) gene expression and/or activity. The small nucleic acid molecules are useful in the treatment of Alzheimer's disease and any other condition that responds to modulation of BACE, APP, pin-1, PS-1 and/or PS-2 expression or activity.06-11-2009
20090149401AMIDE AND PEPTIDE DERIVATIVES OF TETRAALKYLENEPENTAMINES AS TRANSFECTION AGENTS - This invention relates to newly identified pentamine based surfactant compounds, to the use of such compounds and to their production. The invention also relates to the use of the pentamine based compounds to facilitate the transfer of polynucleotides into cells.06-11-2009
20080312176GENE SILENCING - Methods are disclosed for gene silencing (e.g. post transcriptional gene silencing) in an organism using small RNA molecules.12-18-2008
20090176724Methods and Compositions for the Diagnosis, Prognosis and Treatment of Cancer - The invention is relates to splice variants of basal transcription factors and other transcriptional modulators, the use of expression analyses of the same as a diagnostic and prognostic tool, and the targeting of such splice variants for therapeutic purposes, particularly in relation to the treatment of cancer.07-09-2009
20090149413RORS as Modifiers of the p21 Pathway and Methods of Use - Human ROR genes are identified as modulators of the p21 pathway, and thus are therapeutic targets for disorders associated with defective p21 function. Methods for identifying modulators of p21, comprising screening for agents that modulate the activity of ROR are provided.06-11-2009
20090149414rAAV EXPRESSION SYSTEMS AND COMPOSITIONS - Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that are fully virulent, yet lack functional VP2 protein expression. Also disclosed are pharmaceutical compositions, virus particles, host cells, and pharmaceutical formulations that comprise these modified vectors useful in the expression of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in the cells and tissues of selected mammals, including, for example, human tissues and host cells.06-11-2009
20090149406Plasmid PXL3179 or NV1FGF - A prokaryotic recombinant host cell comprising a heterologous replication initiation protein that activates a conditional origin of replication and an extrachromosomal DNA molecule comprising a heterologous therapeutic gene and a conditional origin of replication whose functionality in the prokaryotic recombinant host cell requires a replication initiating protein which is foreign to the host cell is described. The host cell may comprise a pir gene having at least one mutation, which may occur in the pir gene copy number control region, the pir gene leucine zipper-like motif, or the pir gene DNA binding region.06-11-2009
20090149404Oligonucleotide analogues and methods utilizing the same - A method for the prevention or treatment in a mammal of a disease preventable or treatable by the pharmacologically useful antisense or antigene activity of an oligonucleotide analogue or a pharmacologically acceptable salt thereof in the body of said mammal, which method comprises administering to said mammal in need of such prevention or treatment a pharmaceutically effective amount of an oligonucleotide analogue comprising two or more nucleoside units, wherein at least one of said nucleoside units is a structure of the formula (2):06-11-2009
20090149402PHARMACEUTICAL COMPOSITION FOR TREATING CANCER OR DIABETES - A pharmaceutical composition for treating cancer or diabetes which contains the following double-stranded RNA (A) or (B): 06-11-2009
20090018099PROTEIN PRODUCTION - The invention concerns the field of protein production and cell culture technology. CERT is identified as a novel in vivo PKD substrate. Phosphorylation on serine 132 by PKD decreases the affinity of CERT towards its lipid target phosphatidylinositol 4-phosphate at Golgi membranes and reduces ceramide transfer activity, identifying PKD as a regulator of lipid homeostasis. The present invention shows that CERT in turn is critical for PKD activation and PKD dependent protein cargo transport to the plasma membrane. The interdependence of PKD and CERT is thus a key to the maintenance of Golgi membrane integrity and secretory transport.01-15-2009
20080261903Regulation of Kinase, Regulated in Copd Kinase (Rc Kinase) - Reagents which regulate human RC Kinase activity and reagents which bind to human RC Kinase gene products can be used to regulate this protein for therapeutic effects. Such regulation is particularly useful for treating chronic obstructive pulmonary disease, asthma, cancer, and diseases in which cell signaling is defective.10-23-2008
20090093430Biomarkers and methods for identification of agents useful in the treatment of affective disorders - Potential therapeutic, agent for the prevention, treatment or amelioration of an affective disorder, such as a bipolar disorder, depression or anxiety disorder are identified. Also provided are methods for diagnosing and monitoring affective disorders. The methods are based on changes in expression of one or more genes that are differentially expressed in affective disorders.04-09-2009
20090286753NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAS - The invention relates to ribonucleic acids and oligonucleotide probes useful for detection and analysis of microRNAs and their target mRNAs, as well as small interfering RNAs (siRNAs).11-19-2009
20090036392COMPOSITION COMPRISING BIODEGRADABLE HYDRATING CERAMICS FOR CONTROLLED DRUG DELIVERY - The present invention relates to a drug carrier composition comprising i) one or more biodegradable hydrating ceramics ii) one or more expandable agents, and iii) sorbed aqueous medium which in solid form has a ruptured structure. The function of the expandable agent is to create a ruptured structure in the solidified composition, either a foam-like structure or a disintegrated structure where it is split into a large number of parts, particles, units, granules or pieces, so as to obtain an enlarged apparent surface area that is exposed to degradation or erosion upon administration. Suitable substances to obtain this surface enlarging effect are gas-forming agents or swelling agents, gelling agents or disintegrants, here referred to as expandable agents. The expandable agents may be bioresorbable or non-bioresorbable.02-05-2009
20090170794Small interfering rnas that efficiently inhibit viral expression and methods of use thereof - The invention provides methods, compositions, and kits comprising small interfering RNA (shRNA or siRNA) which are useful for inhibition of viralmediated gene expression. Small interfering RNAs as described herein may be used in methods of treatment of HCV infection. ShRNA and siRNA constructs that target the internal ribosome entry site (IRES) sequence of HCV are described.07-02-2009
20090143323COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF A GENE FROM THE EBOLA - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the Ebola virus.06-04-2009
20090143322CELL TRANSFECTING FORMULATIONS OF SMALL INTERFERING RNA RELATED COMPOSITIONS AND METHODS OF MAKING AND USE - Compositions incorporating small interfering ribonucleic acid (siRNA) and certain lipid-conjugated polyamide compound-based delivery vehicles that are particularly useful in the delivery siRNA and other polynucleotides to cells. Also, methods of making and using the compositions.06-04-2009
20090093439RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating chromosomal translocation gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of chromosomal translocation gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of BCR-ABL, ERG, EWS-ERG, TEL-AML1, EWS-FLI1, TLS-FUS, PAX3-FKHR, and/or AML1-ETO fusion genes.04-09-2009
20090088402NOVEL AGENT FOR INDUCING APOPTOSIS COMPRISING MSX1 OR A GENE ENCODING THE SAME AS AN ACTIVE INGREDIENT - The present invention relates to a novel use of Msx1 protein or a nucleotide encoding the same for inducing apoptosis. The Msx1 of the present invention induces apoptosis through direct interaction with p53 via a homeodomain and such interaction leads to increased stability, and/or nuclear localization of p53 in cells. The Msx1 or homeodomain thereof can be effectively used for the treatment of tumors, in which wild-type p53 protein has lost its function by some mechanism that inactivates p53 proteins.04-02-2009
20090170800GENETIC CONSTRUCTS AND COMPOSITIONS COMPRISING RRE AND CTE AND USES THEREOF - Genetic constructs that comprise a coding sequence for HIV-1 Rev, and a coding sequence for a desired protein are disclosed. Compositions that comprise at least two nucleic acid molecules in which at least one nucleic acid molecule comprises a coding sequence for HIV-1 Rev, and at least one nucleic acid molecule comprises a coding sequence for a desired protein are disclosed. In such genetic constructs and compositions comprising nucleic acid molecules, the coding sequence for the desired protein comprises at least a portion of coding sequence for an HIV structural protein that includes an RRE and at least one CTE. Methods of inducing an immune response against an immunogen in an individual, methods of delivering proteins to an individual and methods of producing proteins are also disclosed.07-02-2009
20090088398RECOMBINANT ADENOVIRAL VECTORS AND METHODS OF USE - This invention provides a recombinant adenovirus expression vector characterized by the partial or total deletion of the adenoviral protein IX DNA and having a gene encoding a foreign protein or a functional fragment or mutant thereof. Transformed host cells and a method of producing recombinant proteins and gene therapy also are included within the scope of this invention. Thus, for example, the adenoviral vector of this invention can contain a foreign gene for the expression of a protein effective in regulating the cell cycle, such as p53, Rb, or mitosin, or in inducing cell death, such as the conditional suicide gene thymidine kinase. (The latter must be used in conjunction with a thymidine kinase metabolite in order to be effective.)04-02-2009
20090088400PROSTAGLANDIN E2 MODULATION AND USES THEREOF - Methods, uses, kits and products are described for the prognosis, diagnosis, prevention and treatment of myotronic dystrophy type 1 (DM1), and more particularly for the prognosis, diagnosis, prevention and treatment of the congenital form of myotronic dystrophy type 1 (cDM1), based on changes in/modulation of prostaglandin E04-02-2009
20090291906Oligomeric Compounds And Compositions For Use In Modulation Of Small Non-Coding RNAs - Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.11-26-2009
20090291907Oligomeric Compounds And Compositions For Use In Modulation Of Small Non-Coding RNAs - Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.11-26-2009
20080207546RNA APTAMERS AND METHODS FOR IDENTIFYING THE SAME - RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.08-28-2008
20080207540Method For the Identification of Compounds Modulating Reverse Transport of Cholesterol - The invention relates to methods and compounds which can modulate reverse cholesterol transport in a mammal and to screening methods enabling the selection, identification and/or characterization of compounds which can modulate reverse cholesterol transport. The invention also relates to cells, vectors and genetic constructs which can be used to implement said methods, in addition to pharmaceutical compositions intended for the treatment of atherosclerosis.08-28-2008
20080207543MODIFIED PITUITARY GLAND DEVELOPMENT IN OFFSPRING FROM EXPECTANT MOTHER ANIMALS TREATED WITH GROWTH HORMONE RELEASING HORMONE THERAPY - The intramuscular electroporated injection of a protease-resistant growth hormone-releasing hormone (“GHRH”) cDNA into rat dams at 16 days of gestation resulted in the enhanced long-term growth of the FI offspring. The offspring were significantly heavier by one week of age and the difference was sustained to 10 weeks of age. Consistent with their augmented growth, plasma IGF-I concentration of the FI progeny was increased significantly. The pituitary gland of the offspring was significantly heavier, and contained an increased number of somatotropes (cells producing GH) and lactotrophs (prolactin-secreting cells), and is indicative of an alteration in cell lineages. These unique findings demonstrate that enhanced GHRH expression in pregnant dams can result in intergenerational growth promotion, by altering development of the pituitary gland in the offspring.08-28-2008
20080207549GENES AND POLYPEPTIDES RELATING TO HEPATOCELLULAR OR COLORECTAL CARCINOMA - The present application provides novel human genes WDRPUH and KRZFPUH and PPIL1 whose expression is markedly elevated in a great majority of HCC's and colorectal cancers, respectively, compared to corresponding non-cancerous tissues, as well as novel human gene APCDD1 whose expression is elevated in primary colon cancers and down-regulated in response to the transduction of wild-type APC1 into colon cancer cells. The genes and polypeptides encoded by the genes can be used, for example, in the diagnosis of a cell proliferative disease, and as target for developing drugs against the disease.08-28-2008
20080207548COMPOSITIONS AND METHODS FOR THE THERAPY AND DIAGNOSIS OF BREAST CANCER - Compositions and methods for the therapy and diagnosis of cancer, particularly breast cancer, are disclosed. Illustrative compositions comprise one or more breast tumor polypeptides, immunogenic portions thereof, polynucleotides that encode such polypeptides, antigen presenting cell that expresses such polypeptides, and T cells that are specific for cells expressing such polypeptides. The disclosed compositions are useful, for example, in the diagnosis, prevention and/or treatment of diseases, particularly breast cancer.08-28-2008
20080207556Product and Process for Inhibition of Biofilm Development - Disclosed are compositions and methods for the inhibition of biofilm formation or reduction of existing or developing biofilms in a patient. These methods also inhibit the aggregation of bacteria that form biofilms in the airways. The methods include administering to a subject that has or is at risk of developing biofilms a compound or formulation that inhibits the formation or polymerization of actin microfilaments or depolymerizes actin microfilaments at or proximal to the site of biofilm formation. Such a compound can be administered in combination with a compound or formulation that inhibits the accumulation or activity of cells that are likely to undergo necrosis at or proximal to the site of biofilm formation (i.e., neutrophils). The methods and compositions can further include the use of anti-DNA and/or anti-mucin compounds, as well as other therapeutic compounds and compositions.08-28-2008
20080207552Decoy compositions for treating and preventing brain diseases and disorders - The present invention provides introduction of NF-κB decoy oligodeoxynucleotide into rat cranial nerve through a carotid artery during global brain ischemia. Polymerase chain reaction demonstrated that one hour after global brain ischemia, transfected NF-κB decoy oligodeoxynucleotide effectively suppressed expression of tumor necrosis factor α, interleukin 1β and intracellular adhesion molecule 1 messenger RNAs. Terminal deoxynucleotidyl transferase-mediated deoxyuridine nick-end labeling staining and immunohistochemistry using microtubule-associated protein 2 demonstrated that transfected NF-κB decoy oligodeoxynucleotide significantly attenuated neuronal damage seven days after global brain ischemia. Therapeutic transfection of NF-κB decoy oligodeoxynucleotide during brain ischemia may be effective for attenuation of neuronal damage, suggesting a strategy for protecting the cerebrum from global ischemia.08-28-2008
20080207547COMPOSITIONS AND METHODS FOR THE THERAPY AND DIAGNOSIS OF BREAST CANCER - Compositions and methods for the therapy and diagnosis of cancer, particularly breast cancer, are disclosed. Illustrative compositions comprise one or more breast tumor polypeptides, immunogenic portions thereof, polynucleotides that encode such polypeptides, antigen presenting cell that expresses such polypeptides, and T cells that are specific for cells expressing such polypeptides. The disclosed compositions are useful, for example, in the diagnosis, prevention and/or treatment of diseases, particularly breast cancer.08-28-2008
20080207550IMMUNOMODULATORY POLYNUCLEOTIDES AND METHODS OF USING THE SAME - The invention provides immunomodulatory polynucleotides and methods for immunomodulation of individuals using the immunomodulatory polynucleotides.08-28-2008
20090170798Highly safe intranasally administrable gene vaccines for treating alzheimer's disease - An objective of the present invention is to provide a safe and effective vaccine therapy for Alzheimer's disease. A minus strand RNA viral vector carrying amyloid gene was constructed, and administered intranasally to 24- to 25-months-old APP transgenic mice. The level of serum anti-A 42 antibody was determined and showed to be markedly higher than the control. The results of histological investigation showed that the administration of a vector of the present invention markedly reduced senile plaques in all of the frontal lobe, parietal lobe, and hippocampus. The brain A level was also markedly reduced. Furthermore, the administration of a vector of the present invention did not result in lymphocyte infiltration in the central nervous system.07-02-2009
20090170801METHODS OF TREATMENT OF CARDIOVASCULAR AND CEREBROVASCULAR DISEASES WITH FUCOIDAN - A method for treating an ischemic cardiovascular or cerebrovascular disease comprising administrating to a patient in the need of such treatment a pharmaceutical composition comprising fucoidan.07-02-2009
20090170795Kit of parts designed for implementing an antitumoral or antiviral treatment in a mammal - The present invention relates to a kit of parts comprising a nucleic acid sequence encoding a permease and a drug comprising one nucleobase moiety or a precursor thereof. The present invention further relates to a kit of parts comprising a precursor of a drug comprising a gene coding a permease and a nucleic acid sequence comprising a suicide gene. The present invention also relates to a vector comprising a gene coding a permease and a suicide gene.07-02-2009
20090036393Vesicular monoamine transporter gene therapy in Parkinson's disease - The present invention provides methods and compositions for the therapeutic intervention of Parkinson's disease. More particularly, methods of making and sequestering dopamine are disclosed. Additionally, methods of genetically modifying donor cells by gene transfer for grafting into the central nervous system to treat defective, diseased or damaged cells are disclosed. Methods and compositions for carrying out such gene transfer and grafting are described.02-05-2009
20080287382Oligoribonucleotides and methods of use thereof for treatment of alopecia, acute renal failure and other diseases - The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a human p53 gene. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating a patient suffering from alopecia or acute renal failure or other diseases comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The alopecia may be induced by chemotherapy or radiotherapy, and the patient may be suffering from cancer, in particular breast cancer.11-20-2008
20080274991Nucleic Acids Against Viruses, in Particular Hiv - The invention provides a nucleic acid molecule comprising a sequence that is complementary to a mutant of a genomic nucleic acid sequence of an organism capable of mutating its genome. The genomic nucleic acid sequence preferably comprises a conserved, essential region. Furthermore, the genomic nucleic acid sequence preferably comprises a coding sequence and a DNA/RNA regulatory sequence. A pharmaceutical composition and a gene delivery vehicle comprising a nucleic acid molecule of the invention is also provided, as well as a method for counteracting an organism comprising providing the organism with the nucleic acid molecule. The organism is preferably additionally provided with a nucleic acid sequence complementary to a genomic nucleic acid sequence of the organism.11-06-2008
20080274990Cog47 Protein and S100beta Gene Polymorphism - The invention concerns a Cog11-06-2008
20080274989Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof - The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.11-06-2008
20080274992RECOMBINANT MODIFIED ANKARA VIRAL HIV-1 VACCINES - The field of the present invention relates to novel recombinant MVA vectors encoding one or more HIV-1 immunogens as an HIV-1 vaccine candidate and methods of using same.11-06-2008
20080274996RNAi Probes Targeting Cancer-Related Proteins - RNAi sequences that are useful as therapeutics in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma and melanoma; and Alzheimer's disease. These sequences target clusterin, IGFBP-5, IGFBP-2, both IGFBP-2 and -5 simultaneously, Mitf, and B-raf. The invention further provides for the use of these RNAi sequences in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma and melanoma; and Alzheimer's disease, and a method of treating such conditions through the administration of the RNA molecules with RNAi activity to an individual, including a human individual in need of such treatment.11-06-2008
20080274994CARDIAC MYOSIN LIGHT CHAIN KINASE AND METHODS OF USE - The present disclosure provides a cDNA, protein sequence, and genomic structure of the human cardiac isoform of myosin light chain kinase (cMLCK), and describes mutations in the cMLCK gene that are associated with cardiac dysfunction. Methods are provided for identifying individuals who can harbor mutations in the cMLCK gene, or carry alleles that can predisposed them to cardiac dysfunction. Disclosed also is a significant role for cMLCK in modulating cardiac contractility. The cMLCK protein is shown herein to reduce the amplitude of stretch activation and increase the tension production, a property of muscle which has heretofore had an unknown role in cardiac contraction. Moreover, the cMLCK protein is shown to be regionally distributed in the heart, thereby having differential effects on contractility and stretch activation. Methods herein are provided to exploit this effect of cMLCK, to treat individuals who have or are prone to cardiac dysfunction. In addition, methods are provided to identify agents that modulate cMLCK activity, thereby having potential therapeutic importance in the treatment of cardiac dysfunction.11-06-2008
20080274993Methods and compositions for modulating vascular integrity - The invention provides methods and compositions useful for modulating vascular integrity.11-06-2008
20090170792Peptide ligands - A peptide consisting of or comprising an amino acid sequence selected from a ) Px1X2X3 T [SEQ.ID.NO.:11]; b) PSX4S [SEQ.ID.NO.:2]; C) QX5X6X7Q [SEQ.ID.NO.:3]; d) SX8S [SEQ-ID.NO.:4], in which X1, X2 and X3, which may be the same or different, each represents an amino acid residue; X4 represents an amino acid residue; and x 5 and X7, which may be the same or different, each represents an amino acid residue, X6 represents an amino acid residue having an amide side chain; and X8 represent an amino acid having an aliphatic side chain, which peptide binds to dendritic cells and also to other types of cells. The peptide may be used a target non-viral and viral vectors to such cells.07-02-2009
20090170796Histone Demethylation Mediated By The Nuclear Amine Oxidase Homolog LSD1 - LSD1, a homolog of nuclear amine oxidases, functions as a histone demethylase and transcriptional co-repressor. LSD1 specifically demethylates histone H3 lysine 4, which is linked to active transcription. Lysine demethylation occurs via an oxidation reaction that generates formaldehyde. Importantly, RNAi inhibition of LSD1 causes an increase in H3 lysine 4 methylation and concomitant de-repression of target genes, suggesting that LSD1 represses transcription via histone demethylation. The results thus identify a histone demethylase conserved from 07-02-2009
20090170802METHOD FOR PRODUCING POLYMERS - The invention relates to a method for producing polymers, in particular synthetic nucleic acid double strands of optional sequence, comprising the steps: 07-02-2009
20090170797Hunk, a snfi-related kinase essential for mammary tumor metastasis - This invention relates generally to a novel serine/threonine protein kinase, specifically to hormonally up-regulated, neu-tumor-associated kinase (HUNK); and to the role of HUNK in tumor metastasis, primary tumor development, and the prediction of tumor behavior.07-02-2009
20090170799MODULATION OF C-REACTIVE PROTEIN EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of C-reactive protein. The compositions comprise oligonucleotides, targeted to nucleic acid encoding C-reactive protein. Methods of using these compounds for modulation of C-reactive protein expression and for diagnosis and treatment of disease associated with expression of C-reactive protein are provided.07-02-2009
20090170793ARTIFICIAL RIBOSWITCH FOR CONTROLLING PRE-MRNA SPLICING - The present invention relates to riboswitches that have been engineered to regulate pre-mRNA splicing. In particular, the insertion of a high affinity theophylline binding aptamer into the 3′ splice site region, 5′ splice site region, or branchpoint sequence (BPS) of a pre-mRNA modulates RNA splicing in the presence of theophylline. Accordingly, the aspects of the present invention include, but are not limited to, theophylline-dependent riboswitches which modulate RNA splicing, methods of modulating RNA splicing using theophylline and its corresponding riboswitches, methods of improving/identifying theophylline-dependent riboswitches, methods of treating diseases associated with or caused by abnormal RNA splicing.07-02-2009
200802808462', 3'-DIDEOXYNUCLEOSIDE ANALOGUES FOR THE TREATMENT OR PREVENTION OF FLAVIVIRIDAE INFECTIONS - A method for the treatment or prevention of Flaviviridae infections, in particular, hepatitis C virus infection, in a host, and in particular, a human, is provided that includes administering an effective amount of a β-L- or β-D-2′,3′-dideoxynucleoside or a pharmaceutically acceptable salt or prodrug thereof, optionally in a pharmaceutically acceptable diluent or excipient.11-13-2008
20080280843Methods and kits for linking polymorphic sequences to expanded repeat mutations - Methods and kits are provided for determining which single nucleotide polymorphism (“SNP”) variant of an allele of a heterozygous patient is on the same mRNA transcript as a disease-causing mutation that is at a remote region of the gene's mRNA comprising a) an allele-specific reverse transcription reaction using an allele-specific primer, and b) analysis of the resulting cDNA product from the reverse transcription reaction at the region of the mutation to determine the presence or absence of the mutation on this allele-specific cDNA product.11-13-2008
20080280848Structures of Active Guide Rna Molecules and Method of Selection - The present invention relates to methods and compositions for modulating RNA silencing efficiency by providing selective RISC (RNA-induced silencing complex) formation. The present invention also relates to methods for identifying nucleic acids and/or determining their structures.11-13-2008
20080287386Oligoribonucleotides and methods of use thereof for treatment of fibrotic conditions and other diseases - The invention relates to a double-stranded compound, preferably an oligoribonucleotide (siRNA), which down-regulates the expression of a human TGaseII gene at the post-transcriptional level. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating a patient suffering from a fibrotic disease such as pulmonary, kidney and liver fibrosis or ocular, scarring comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The invention also relates to treatment of fibrotic and other diseases by use of antibodies to TGaseII polypeptide.11-20-2008
20080287380Antisense Oligonucleotides Against Cpla2, Compositions and Uses Thereof - Antisense oligonucleotides against cPLA11-20-2008
20080287387Method for Producing Sterile Polynucleotide Based Medicaments - The present invention relates to a novel method for producing formulations comprising a polynucleotide, block copolymer and cationic surfactant. The formulations produced by the current method are suitable for use in polynucleotide based medicaments. A suitable method of production disclosed herein additionally comprises cold filtering a mixture of a polynucleotide, block copolymer and cationic surfactant, thereby sterilizing the formulation. The method of the present invention also eliminates the need for thermal cycling of the formulation, thereby reducing the time and expense required to produce large quantities of a formulation during commercial manufacturing. The present invention also relates to novel cationic lipids.11-20-2008
20080287389Nucleoside analogs in combination therapy of herpes simplex infections - A pharmaceutical product comprising a nucleoside analogue active against herpes simplex virus, such as acyclovir/valaciclovir or peniclovir/famciclovir, and an immunosuppressant, as a combined preparation for simultaneous, separate or sequential use in the treatment and/or prevention of herpes simplex virus infections.11-20-2008
20080287383NUCLEIC ACID COMPOUNDS FOR INHIBITING ERBB GENE EXPRESSION AND USES THEREOF - The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing ERBB gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an ERBB mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an ERBB gene in a cell or in a subject to treat an ERBB-related disease.11-20-2008
20080287381Injectable Agent for the Targeted Treatment of Retinal Ganglion Cells - The present invention relates to the targeted treatment of retinal ganglion cells (RGC).11-20-2008
20080287379Oligonucleotide Complex Compositions and Methods of Use as Gene Alteration Tools - Compositions and methods of treatments of cells are provided for altering the phenotype of a cell by administering an oligonucleotide complex to the cell, the complex having two strands and chemical modifications.11-20-2008
20080293659FC gamma RIIA-specific nucleic acid interference - The present invention provides methods and compositions for attenuating expression of FcγRIIA. In general, the described methodology involves the use of RNAi constructs that are targeted to a FcγRIIA mRNA sequence.11-27-2008
20080293652Combination of Ad-P53 and Chemotherapy for the Treatment of Tumours - The present invention relates to the use of p53 gene therapy to treat recurrent cancers in combination with a radio- or chemotherapy. Patients with recurring cancers are treated with a p53 expression construct followed by subsequent radio- or chemotherapy treatment. Viral and non-viral delivery systems, as well as various radio- and chemotherapy regimens are disclosed.11-27-2008
20080293658ANTISENSE OLIGONUCLEOTIDES CAPABLE OF INHIBITING THE FORMATION OF CAPILLARY TUBES BY ENDOTHELIAL CELLS - A pharmaceutical composition that blocks angiogenesis comprising as active agent at least one substance selected from the group consisting of (i) a nucleic acid molecule of a gene coding for protein IRS-1, a complementary sequence or a fragment thereof and (ii) a molecule which inhibits expression of a nucleic acid molecule according to (i).11-27-2008
20080293657MODULATION OF THE EXPRESSION OF STAT-1-DEPENDENT GENES - The present invention relates to decoy-oligonucleotides and antisense-oligonucleotides having the nucleic acid sequence according to SEQ ID NO: 1 to 43 as well as the use thereof as medicaments.11-27-2008
20080306016Nucleic Acid Functionalized Nanoparticles for Therapeutic Applications - Materials and Methods for modulating cellular uptake of functionalized nanoparticles are provided. Also provided are materials and methods for modulating the effectiveness of a therapeutic agent with a functionalized nanoparticle.12-11-2008
20080306015siRNA targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to PCSK9.12-11-2008
20080306018Micro-Rna Expression Abnormalities of Pancreatic, Endocrine and Acinar Tumors - The present invention provides novel methods and compositions for the diagnosis, prognosis and treatment of pancreatic cancer. The invention also provides methods of identifying anti-pancreatic cancer agent.12-11-2008
20080306017Microrna-Based Methods and Compositions for the Diagnosis, Prognosis and Treatment of Lung Cancer - The present invention provides novel methods and compositions for the diagnosis, prognosis and treatment of lung cancer. The invention also provide methods of identifying anti-lung cancer agents.12-11-2008
20080312177SERCA2 THERAPEUTIC COMPOSITIONS AND METHODS OF USE - The present invention provides methods for treating urinary incontinence, urethral sphincter dysfunction and/or bladder dysfunction by delivering a therapeutic adeno-associated virus (AAV)-SERCA2 composition to a subject in need thereof.12-18-2008
20080312175METHODS AND COMPOSITIONS FOR REGULATING HDAC4 ACTIVITY - The present invention provides methods and compositions for modulating HDAC4 activity and modulating the activity of proteins downstream of HDAC4 in the muscle transcriptional pathway in a cell by modulating HDAC4 activity. Further provided are methods and compositions for treating muscle atrophy and/or inflammation by inhibiting HDAC4 activity.12-18-2008
20080312171Tetracycline-Dependent Regulation of Rna Interference - The present invention relates to a tetracycline dependent gene regulatory system or composition controlling the expression of a target gene in a cell and to methods using said system or composition. The present invention more specifically discloses compositions, vectors and methods allowing tetracycline-controlled expression of short-hairpin RNAs (shRNAs), and demonstrates inducible, reversible and stable RNA interference (RNAi) using the same in a cell. The invention can be used to cause reversible control of the expression of any gene and may therefore find applications in the fields of mammalian, in particular human, genetics and molecular therapeutics, in cell and gene therapy, research as well as in genetic studies using transgenic animals.12-18-2008
20080312170Fibrinolysin of Agkistrodon Acutus Venom and its Usage - The present invention relates to a fibrinolysin gene of Agkistrodon acutus, a vector containing the said gene and the host cell which transformed by the vector. The fibrinolysin could be used in treating diseases caused by thrombus. The fibrinolysin FII is purified from the crude venom of Agkistrodon acutus, and the corresponding gene is cloned. It is recommended to produce the fibrionolysin by the yeast expression system. The activity of the fibrionlysin is also determined. The advantages of this invention are that the expression level and activity of the fibrinolysin are both high and the quality of the fibrinolysin is stable.12-18-2008
20080312172Anti-IL-6 Antibodies, Compositions, Methods and Uses - The present invention relates to at least one novel chimeric, humanized or CDR-grafted anti-IL-6 antibodies derived from the murine CLB-8 antibody, including isolated nucleic acids that encode at least one such anti-IL-6 antibody, vectors, host cells, transgenic animals or plants, and methods of making and using thereof, including therapeutic compositions, methods and devices.12-18-2008
20080269149Chimeric Vectors - The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent.10-30-2008
20080312178Human G-Protein Receptor HGBER32 - Human G-protein coupled receptor polypeptides and DNA (RNA) encoding such polypeptides and a procedure for producing such polypeptides by recombinant techniques is disclosed. Also disclosed were methods for utilizing such polypeptides for identifying antagonists and agonists to such polypeptides and methods of using the agonists and antagonists therapeutically to treat conditions related to the underexpression and overexpression of the G-protein coupled receptor polypeptides, respectively. Also disclosed are diagnostic methods for detecting a mutation in the G-protein coupled receptor nucleic acid sequences and an altered level of the soluble form of the receptors.12-18-2008
20080200414HCaRG, A Novel Calcium-Regulated Gene - Novel nucleic acids and corresponding encoded proteins are described. Also described are corresponding recombinant vectors and host cells, as well as methods of producing the proteins. Also described are mimetics and antibodies to the proteins as well as compositions comprising the nucleic acid or proteins or a portion thereof. Methods and kits for the detection of a disease, disorder or abnormal physical state caused by abnormal modulation of calcium levels in a patient are also described. Methods for treating a patient having a disease, disorder or abnormal physical state caused by abnormal calcium levels are also described. Methods for assaying abnormal calcium levels are also described, as are methods for screening the efficacy of products for modulating abnormal calcium levels.08-21-2008
20080293654Therapeutic methods using Smads - Methods of inducing the expression of a Smad in a cell or tissue comprising the step of contacting the cell or tissue capable of expressing the Smad with a bone morphogenic protein are provided. Methods of inducing tissue formation and repairing a tissue defect or regenerating tissue, at a target locus in a mammal comprising the step of administering to the target locus a Smad are also provided.11-27-2008
20080207545Methods and Compositions for Treating 5Alpha-Reductase Type 1 and Type 2 Dependent Conditions - The invention relates generally to the use of anti-sense oligonucleotides, small interfering RNA, and ribozymes to modulate expression of the human steroid 5α-reductase gene and thereby modulate levels of dihydrotestosterone (DHT). Elevated levels of DHT are associated with various disorders including, but not limited to, skin diseases, hair loss, hirsuitism, and benign prostatic hyperplasia. The invention specifically relates to formulations of these anti-sense oligonucleotides, small interfering RNA, and ribozymes for administration to treat and prevent disorders08-28-2008
20080207539Self-Processing Rna Expression Cassette - The invention provides a self-processing RNA expression cassette which includes at least one pair of processing units, an RNAi effecter sequence of predetermined length that regulates target gene expression which is flanked by said pair of processing units; and at least one pair of cognate ribozyme cis-cleavage target sites located 5′ and 3′ of the RNAi effecter sequence. The self-processing RNA expression cassette is able to express in vivo and in vitro and the RNAi effecter sequence includes at least one target recognition sequence derived from the Hepatitis B Virus (HBV) X gene (HBx).08-28-2008
20080287388Human G-Protein Coupled Receptor - A human G-protein coupled receptor polypeptide and DNA (RNA) encoding such polypeptide and a procedure for producing such polypeptide by recombinant techniques is disclosed. Also disclosed are methods for utilizing such polypeptide for identifying antagonists and agonists to such polypeptide. Antagonists and agonists may be used therapeutically to inhibit or stimulate the G-protein coupled receptor. Also disclosed are assays for detecting mutations in the nucleic acid sequence encoding the G-protein coupled receptor.11-20-2008
20080242632MULTI-TARGETING SHORT INTERFERING RNAs - The present invention relates to novel short interfering RNA (siRNA) molecules that are multi-targeted. More specifically, the present invention relates to siRNA molecules that target two or more sequences. In one embodiment, multi-targeting siRNA molecules are designed to incorporate features of siRNA molecules and features of micro-RNA (miRNA) molecules. In another embodiment, multi-targeting siRNA molecules are designed so that each strand is directed to separate targets.10-02-2008
20080242623Microutrophin and Uses Thereof - A microutrophin containing a utrophin having internal deletions (relative to a native utrophin) in the hinge regions and a C-terminal deletion is provided. Also provided are vectors and compositions useful for delivering the microutrophin for the treatment of muscular disorders, including Duchenne Muscular Dystrophy.10-02-2008
20080293653Method of treating autoimmune diseases - The invention features a method of inducing an apoptosis-resistant cell to undergo apoptosis, in which the cell is associated with an autoimmune disease such as multiple sclerosis. The method involves sensitizing the cell to apoptosis stimuli by treating the cell with an IAP antisense oligonucleotide, so that the cell undergoes apoptosis at a site of autoimmune disease.11-27-2008
20090042827ANTIVIRAL OLIGONUCLEOTIDES TARGETING HBV - Random sequence oligonucleotides that have antiviral activity are described, along with their use as antiviral agents. In many cases, the oligonucleotides are greater than 40 nucleotides in length. Also described are methods for the prophylaxis or treatment of a viral infection in a human or animal, and a method for the prophylaxis treatment of cancer caused by oncoviruses in a human or animal. The methods typically involve administering to a human or animal in need of such treatment, a pharmacologically acceptable, therapeutically effective amount of at least oligonucleotide that does not act by a sequence complementary mode of action.02-12-2009
20080207553Biodegradable Cationic Polymers - Polymers comprising a polyethylenimine, a biodegradable group, and a relatively hydrophobic group are useful for the delivery of bioactive agents to cells.08-28-2008
20080269154Novel neurotrophic factor protein and uses thereof - The present invention discloses a novel neurotrophic factor protein, MANF2 and a genetic sequence encoding the same. The molecule will be useful in the development of a range of therapeutics and diagnostics useful in the treatment, prophylaxis and/or diagnosis of MANF2 dependent conditions. The molecule of the present invention is also a useful effector of primary and central neurons, especially dopaminergic neurons at the central nervous system and growth factor genes.10-30-2008
20080269151Fusion proteins of mycobacterium tuberculosis - The present invention relates to fusion proteins containing at least two 10-30-2008
20080269148Modified Small Interfering Rna Molecules and Methods of Use - The present invention provides double-stranded RNA molecules that mediate RNA interference in target cells, preferably hepatic cells. The invention also provides double-stranded RNA (dsRNA) molecules that are modified to be resistant to nuclease degradation, which inactivates a virus, and more specifically, hepatitis C virus (HCV). The invention also provides a method of using these modified RNA molecules to inactivate virus in mammalian cells and a method of making modified small interfering RNAs (siRNAs) using human Dicer. The invention provides modified RNA molecules that are modified to include a dsRNA or siRNA wherein one or more of the pyrimidines in the RNA molecule are modified to include 2′-Fluorine. The invention also provides dsRNA or siRNA in which all pyrimidines are modified to include a 2′-Fluorine. The invention provides that the 2′-Fluorine dsRNA or siRNA molecule is further modified to include a two base deoxynucleotide “TT” sequence at the 3′end of the molecule.10-30-2008
20080269153INCREASED STABILITY OF A DNA FORMULATION BY INCLUDING POLY-L-GLUTAMATE - Aspects of the present invention is related to DNA vaccine formulations having enhanced stability comprising at least one DNA plasmid capable of expressing an antigen in cells of mammal and poly-L-glutamate; wherein the DNA plasmid is present in the vaccine formulation at a concentration of at least 1 mg/ml, and the poly-L-glutamate is present in the amount of weight that is 1% of the amount of DNA plasmid. Some aspects of the present invention is related to methods of stabilizing DNA plasmid in a DNA vaccine formulation. Additionally, the present invention is related to methods for introducing a DNA vaccine formulation having enhanced stability into a cell of a selected tissue in a recipient.10-30-2008
20080269159COMPOSITIONS AND METHODS FOR REGULATION OF SMOOTH MUSCLE CELLS AND BLOOD PRESSURE - The invention encompasses a composition for regulating smooth muscle cells. In particular, the invention encompasses a vector comprising a smooth muscle promoter operably-linked to a nucleic acid encoding a calcium-activated potassium channel.10-30-2008
20080269155REMEDY OR PREVENTIVE FOR KIDNEY DISEASE AND METHOD OF DIAGNOSING KIDNEY DISEASE - A novel agent for therapy and/or prevention of kidney diseases as well as a diagnostic method (detection method) of kidney diseases is disclosed. The agent for therapy and/or prevention of kidney diseases comprises as an effective ingredient a substance which inhibits casein kinase 2. The diagnostic method of kidney diseases according to the present invention comprises measuring activity or content of casein kinase 2, or measuring expression amount of casein kinase 2 gene in a sample separated from body.10-30-2008
20080269147RNA interference mediating small RNA molecules - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a 10-30-2008
20090264381NUCLEIC ACID AND CORRESPONDING PROTEIN ENTITLED 151P3D4 USEFUL IN TREATMENT AND DETECTION OF CANCER - A novel gene (designated 151P3D4) and its encoded protein, and variants thereof, are described wherein 151P3D4 exhibits tissue specific expression in normal adult tissue, and is aberrantly expressed in the cancers listed in Table I. Consequently, 151P3D4 provides a diagnostic, prognostic, prophylactic and/or therapeutic target for cancer. The 151P3D4 gene or fragment thereof, or its encoded protein, or variants thereof, or a fragment thereof, can be used to elicit a humoral or cellular immune response; antibodies or T cells reactive with 151P3D4 can be used in active or passive immunization.10-22-2009
20090099124SHORT INTERFERING RNA AS AN ANTIVIRAL AGENT FOR HEPATITIS C - Hepatitis C virus (HCV) is a major cause of chronic liver disease and affects over 270 million individuals worldwide. The HCV genome is a single-stranded RNA that functions as both a messenger RNA and replication template, making it an attractive target for the study of RNA interference. Double-stranded short interfering RNA (siRNA) molecules designed to target the HCV genome are disclosed herein.04-16-2009
20090181907Drug Comprising As The Active Ingredient Proliferative Vector Containing Survivin Promoter - It is intended to provide a drug to be used in gene therapy which specifically targets abnormal cells such as tumor cells and destroys the same for healing. Namely, a drug comprising, as the active ingredient, a proliferative vector which contains a Survivin promoter proliferating depending on the expression of Survivin. This drug may be used in order to treat tumor. In this drug, use may be made of an adenovirus as the vector. In the adenovirus of this drug, an endogenous promoter of an E1A domain may be substituted with a Survivin promoter.07-16-2009
20090124570Methods and Compositions for Facilitating Tissue Repair and Diagnosing, Preventing, and Treating Fibrosis - Compositions and methods for facilitating tissue repair by enhancing the actions of BIGH3 or at least one of its downstream effector molecules in injured tissue, and for the diagnosis, prophylactic and therapeutic treatment of fibrosis by inhibiting the actions of BIGH3 or at least one of its downstream effector molecules, such as PU.1 transcription factor and MMP14. Other disclosed methods include methods of screening and/or identifying compounds useful for facilitating tissue repair, treating fibrosis, or for altering the accumulation or deposition of collagen, comprising contacting BIGH3 or its downstream effector molecules, such as PU.1 or MMP14, with a substance and subsequently determining the effects of the substance on the activity of BIGH3, PU.1, or MMP14.05-14-2009
20090186848ANTISENSE ANTIVIRAL COMPOUNDS AND METHODS FOR TREATING A FILOVIRUS INFECTION - The invention provides antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Filoviridae family, and in the treatment of a viral infection. The compounds and methods relate to the treatment of viral infections in mammals including primates by Ebola and Marburg viruses. The antisense antiviral compounds are morpholino oligonucleotides having: a) a nuclease resistant backbone, b) 15-40 nucleotide bases, and c) a targeting sequence of at least 15 bases in length that hybridizes to a target region selected from the following: i) the Ebola virus AUG start site region of VP24; ii) the Ebola virus AUG start site region of VP35; iii) the Marburg virus AUG start site region of VP24; or iv) the Marburg virus AUG start site region of NP.07-23-2009
20090029929Decoy Nucleic Acid to Synoviolin Gene Promoter - The present invention provides a decoy nucleic acid for the Synoviolin gene promoter. Also provided is the decoy nucleic acid can inhibit the promoter activity by binding to the transcription factor of the Synoviolin gene promoter. It also provides a decoy nucleic acid as expressed in (a) a decoy nucleic acid consisting of a nucleotide sequence as shown in SEQ ID NO: 11 or 12; and it also provides (b) a decoy nucleic acid consisting of a nucleotide sequence as shown in SEQ ID NO: 11 or 12 having deletion, substitution or addition of one or several nucleotides and has the function of inhibiting Synoviolin gene promoter activity.01-29-2009
20090029934RNA interference in respiratory epithelial cells - The present invention is directed to small interfering RNA molecules targeted against a gene of interest in respiratory epithelial cells, and methods of using these RNA molecules.01-29-2009
20090029933INHIBITOR OF PEROXISOME PROLIFERATOR-ACTIVATED RECEPTOR ALPHA COACTIVATOR 1 - The present invention refers to the use of an antisense DNA oligonucleotide for the messenger RNA of the PGC-1α protein, useful as drug for the treatment of diabetes mellitus, insulin resistance and metabolic syndrome. More specifically, the present invention deals with a compound used as drug, through enteral or parenteral route, preferably, with the property of inhibiting the protein expression peroxisome proliferator-activated receptor alpha Coactivator 1 (PGC-1α) leading to the reduction of the blood glucose levels. It deals, therefore, with a pharmacological compound that promotes, in diabetic and insulin-resistant individuals, improvement of the glucose serum levels, increase of the plasmatic insulin concentration and reduction of insulin resistance. The present invention presents a more effective control of the glucose levels and acts beneficially on other complications associated to the Diabetes and obesity conditions, according to tests performed in animal models. In this manner, the principal advantage of the present invention over others alike already existing in the market is the effectiveness that controls blood glucose levels and the fact of acting beneficially on other complications that accompany the disease.01-29-2009
20090029931Topical administrations of antisense compounds to vla-4 for the treatment of respiratory conditions - A method for the treatment and/or prophylaxis of an animal having a respiratory disease or condition associated with airway hyperresponsiveness, eosinophilia, neutrophilia, leukocytes or overproduction of mucus and/or with the expression of integrin α4 comprising administering to the animal a composition comprising from. 0.001 to 1000 μg per kg body weight of the animal of an antisense compound targeted to a nucleic acid molecule encoding integrin α4.01-29-2009
20090088399Use of Light Sensitive Genes - The invention relates to the use of a light-gated ion channel for the manufacture of a medicament for the treatment of blindness and a method for expressing said cell specific fashion, e.g. in ON-bipolar cells, ON-ganglion cells, or AII amacrine cells.04-02-2009
20090181908METHODS AND COMPOSITIONS FOR TREATING KERATIN HYPERPROLIFERATIVE DISORDERS - A method for keratin hyperproliferation disorders such as corns, calluses, or keratosis pilaris (KP) by administering to a subject experiencing the disorder a therapeutically effective amount of an RNA sequence which inhibits expression of a gene encoding for a keratin selected from the group consisting of K6a, K6b, K16, K17, and combinations thereof.07-16-2009
20090181912GLP/1/EXENDIN 4 IgG Fc FUSION CONSTRUCTS FOR TREATMENT OF DIABETES - The invention is a method and composition for the prevention and treatment of type I and type II diabetes in a subject. The composition comprises an IgG-Fc fusion protein where the fusion protein comprises GLP-1, mutant GLP-1, or exendin-4.07-16-2009
20090181911Role of gax in alzheimer neurovascular dysfunction - Neurovascular disorder critically contributes to the development and pathogenesis of Alzheimer's disease (AD). Transcriptional profiling of human brain endothelial cells (BEC) defines a subset of age-independent genes significantly altered in AD including the homebox gene GAX whose expression controls vascular phenotype and is low in AD. By using viral-mediated GAX gene silencing and transfer, restoring GAX expression in AD BEC is angio-genic, transcriptionally suppresses the AFX1 forkhead transcription factor-mediated apoptosis, and increases the levels of a major amyloid β-peptide (Aβ) clearance receptor, the low density lipoprotein receptor-related protein 1 (LRP-1) at the blood-brain barrier. In a mouse model of Alzheimer's disease, deletion of the Gax gene results in reductions in brain capillary density and the resting cerebral blood flow, loss of angiogenic brain response to hypoxia, and an impaired Aβ brain efflux caused by reduced LRP-1 levels. The link of GAX gene to AD neurovascular dysfunction provides new mechanistic and therapeutic insights into AD.07-16-2009
20080269160INDUCED ACTIVATION IN DENDRITIC CELLS - The present invention is directed to a composition and method which to treat diseases and to enhance a regulated immune response. More particularly, the present invention is drawn to compositions that are based on dendritic cells modified to express an inducible form of a co-stimulatory polypeptide.10-30-2008
20090082303DRUG FOR PREVENTING AND TREATING ATHEROSCLEROSIS - The present invention relates to a prophylactic/therapeutic agent for atherosclerosis comprising GM3 synthase inhibitor or inhibitor for expression of GM3 synthase gene, a diagnostic product for atherosclerosis comprising antibody to GM3 synthase, a method of diagnosis for atherosclerosis using antibody to GM3 synthase and others, an use of GM3 or GM3 synthase for diagnostic marker of atherosclerosis, and the like.03-26-2009
20090137508RNA INTERFERENCE MEDIATED INHIBITION OF POLYCOMB GROUP PROTEIN EZH2 GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating polycomb group protein EZH2 gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of polycomb group protein EZH2 gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of polycomb group protein EZH2 genes, such as EZH2.05-28-2009
20090137509RNA INTERFERENCE MEDIATED INHIBITION OF PROLIFERATION CELL NUCLEAR ANTIGEN (PCNA) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating PCNA gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of PCNA gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of PCNA genes. The small nucleic acid molecules are useful in the treatment of cancer or restenosis or other proliferative diseases, disorders, or conditions.05-28-2009
20090137500RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.05-28-2009
20090137503Pharmacological modulation of telomere length in cancer cells for prevention and treatment of cancer - Acyclic nucleoside analogs such as acyclovir, ganciclovir, penciclovir and the corresponding pro-drugs, i.e., valacyclovir, valganciclovir and famciclovir, respectively have been identified as inhibitors or antagonists of both telomerase (encoded by TERT) and reverse transcriptase encoded by L-1 (LINE-1) RT, and as useful for treating or preventing cancers induced or mediated by the two enzymes. Method of treating or preventing such cancers in patients involves administration of a therapeutically effective amount of a composition having an inhibitor or antagonist of the reverse transcriptases in cells of the patients. The inhibitor or antagonist blocks lengthening of telomeres in telomerase positive and telomerase negative cells. Methods and kits for detecting pathologically proliferating cells expressing TERT and L1RT are also disclosed.05-28-2009
20090181909Vasopressin-Binding L-Nucleic Acid - The invention relates to a vasopressin-binding nucleic acid, characterized in that the nucleic acid has a Box 07-16-2009
20090163433ANTHELMINTIC AND/OR INSECTICIDE DEVELOPMENT - The use of a nucleic acid molecule encoding FAS in a nematode or arthropod, or a fragment or variant thereof, to identify or produce FAS as a target for: endectocide; anthelmintic and/or insecticide; development.06-25-2009
20090163429RNA APTAMERS AND METHODS FOR IDENTIFYING THE SAME - RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.06-25-2009
20090163430FUNCTIONS AND TARGETS OF LET-7 MICRO RNAS - The present invention concerns methods and compositions for treating or assessing treatment of diseases related to mis-expression of genes or genetic pathways that can be modulated by let-7. Methods may include evaluating patients for genes or genetic pathways modulated by let-7, and/or using an expression profile to assess the condition of a patient or treating the patient with an appropriate miRNA.06-25-2009
20090143320Kinase suppressor of Ras inactivation for therapy of Ras mediated tumorigenesis - The present invention relates to methods and compositions for the specific inhibition of kinase suppressor of Ras (KSR). In particular, the invention provides genetic approaches and nucleic acids for the specific inhibition of KSR, particularly of KSR expression. The invention relates to antisense oligonucleotides and the expression of nucleic acid which is substantially complementary to KSR RNA. Oligonucleotide and nucleic acid compositions are provided. The invention provides methods to inhibit KSR, including inhibition of KSR expression. Methods for blocking gf Ras mediated tumorigenesis, metastasis, and for cancer therapy are provided. Methods for conferring radiosensitivity to cells are also provided.06-04-2009
20090186849ANTISENSE ANTIVIRAL COMPOUNDS AND METHODS FOR TREATING A FILOVIRUS INFECTION - The invention provides antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Filoviridae family, and in the treatment of a viral infection. The compounds and methods relate to the treatment of viral infections in mammals including primates by Ebola and Marburg viruses. The antisense antiviral compounds are morpholino oligonucleotides having: a) a nuclease resistant backbone, b) 15-40 nucleotide bases, and c) a targeting sequence of at least 15 bases in length that hybridizes to a target region selected from the following: i) the Ebola virus AUG start site region of VP24; ii) the Ebola virus AUG start site region of VP35; iii) the Marburg virus AUG start site region of VP24; or iv) the Marburg virus AUG start site region of NP.07-23-2009
20090186847ANTISENSE ANTIVIRAL COMPOUNDS AND METHODS FOR TREATING A FILOVIRUS INFECTION - The invention provides antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Filoviridae family, and in the treatment of a viral infection. The compounds and methods relate to the treatment of viral infections in mammals including primates by Ebola and Marburg viruses. The antisense antiviral compounds are morpholino oligonucleotides having: a) a nuclease resistant backbone, b) 15-40 nucleotide bases, and c) a targeting sequence of at least 15 bases in length that hybridizes to a target region selected from the following: i) the Ebola virus AUG start site region of VP24; ii) the Ebola virus AUG start site region of VP35; iii) the Marburg virus AUG start site region of VP24; or iv) the Marburg virus AUG start site region of NP.07-23-2009
20090069264TOXOPLASMA GONDII NUCLEIC ACID MOLECULES - The present invention relates to immunogenic 03-12-2009
20090137515ABUSE-RESISTANT AMPHETAMINE PRODRUGS - The invention describes compounds, compositions, and methods of using the same comprising a chemical moiety covalently attached to amphetamine. These compounds and compositions are useful for reducing or preventing abuse and overdose of amphetamine. These compounds and compositions find particular use in providing an abuse-resistant alternative treatment for certain disorders, such as attention deficit hyperactivity disorder (ADHD), ADD, narcolepsy, and obesity. Oral bioavailability of amphetamine is maintained at therapeutically useful doses. At higher doses bioavailability is substantially reduced, thereby providing a method of reducing oral abuse liability. Further, compounds and compositions of the invention decrease the bioavailability of amphetamine by parenteral routes, such as intravenous or intranasal administration, further limiting their abuse liability.05-28-2009
20090137501Resistance Genes - Genes involved in immune resistance to infection and uses thereof are described. In particular genes which are involved in resistance to HIV infection and in slowing disease progression in infected individuals are described.05-28-2009
20090137519SEMI-SOFT C-CLASS IMMUNOSTIMULATORY OLIGONUCLEOTIDES - The invention relates to specific C-Class semi-soft CpG immunostimulatory oligonucleotides that are useful for stimulating an immune response. In particular the oligonucleotides are useful for treating allergy, such as allergic rhinitis and asthma, cancer and infectious disease, such as hepatitis B and hepatitis C.05-28-2009
20090137516COMPOSITIONS AND METHODS OF TREATING DYSLIPIDEMIA - The invention relates to the treatment of subjects for the purpose of reducing serum LDL, VLDL, triglycerides and fatty acids, by administering agents which reduce the activity of the bile acid pathway component SHP. Methods and pharmaceutical preparations comprising such agents are provided.05-28-2009
20090137502Riproximin, a Novel Type II Ribosome-Inactivating Protein and Uses Thereof - Described is a novel type II ribosome-inactivating protein, riproximin, as well as nucleic acid molecules encoding said protein. Furthermore, therapeutic uses of riproximin are described.05-28-2009
20090118209Use of the slug gene as a genetic marker in functions mediated by SCF (stem cell factor) and applications - The Slug gene mediates the functions of SCF linking and its c-kit receptor which means that the Slug gene, the Slug gene's cDNA, Slug protein and/or drugs or substances that activate the expression of the Slug gene can be used as therapeutic agents in the mobilization of hematopoyetic stem cells for transplants or gene therapy, in the ex vivo expansion of hematopoyetic stem cells and/or in the treatment of masculine sterility problems.05-07-2009
20090118208Composition and methods of RNAI therapeutics for treatment of cancer and other neovascularization diseases - Compositions and methods are provided for treatment of diseases involving unwanted neovascularization (NV). The invention provides treatments that control NV through selective inhibition of pro-angiogenic biochemical pathways, including inhibition of the VEGF pathway gene expression and inhibition localized at pathological NV tissues. Tissue targeted nanoparticle compositions comprising polymer conjugates and nucleic acid molecules that induce RNA interference (RNAi) are provided. The nanoparticle compositions of the invention can be used alone or in combination with other therapeutic agents such as VEGF pathway antagonists. The compositions and methods can be used for the treatment of NV diseases such as cancer, ocular disease, arthritis, and inflammatory diseases.05-07-2009
20090005336Use of the microRNA miR-1 for the treatment, prevention, and diagnosis of cardiac conditions - Among >300 miRNAs known to date, miR-1 is considered muscle-specific. Here we show that that miR-1 overexpressed in individuals with coronary artery disease, and when overexpressed, it exacerbated arrhythmogenesis in both infarcted and normal hearts of rats whereas elimination of miR-1 by its antisense inhibitor relieved it. MiR-1 rendered slowed conduction and depolarized membrane by post-transcriptionally repressing KCNJ2 and GJA1 genes, likely accounting for its arrhythmogenic potential. Thus, miR-1 may have important pathophysiological functions in heart, being a novel antiarrhythmic target useful in the treatment and prevention of various cardiac pathologies.01-01-2009
20090005333REDUCING CULLING IN HERD ANIMALS GROWTH HORMONE RELEASING HORMONE (GHRH) - One aspect of the current invention is a method of decreasing an involuntary cull rate in farm animals, wherein the involuntary cull results from infection, disease, morbidity, or mortality. Additionally, milk production, animal welfare, and body condition scores are improved by utilizing methodology that administers the isolated nucleic acid expression construct encoding a GHRH or functional biological equivalent to an animal through a parenteral route of administration. Following a single dose of nucleic acid expression vector, animals are healthier and effects are demonstrated long term without additional administration(s) of the expression construct.01-01-2009
20090005332Compositions and Methods for Modulating Gene Expression Using Self-Protected Oligonucleotides - The present invention is directed to novel nucleic acid molecules which include a region complementary to a target gene and one or more self-complementary regions, and the use of such nucleic acid molecules and compositions comprising the same to modulate gene expression and treat a variety of diseases and infections.01-01-2009
20090176723Methods and compositions involving miRNA and miRNA inhibitor molecules - The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.07-09-2009
20090186843RNA sequence-specific mediators of RNA interference - The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.07-23-2009
20090186842Methods and compositions for inhibition of viral replication - The present invention is directed to methods and compositions that are effective in the inhibition of viral replication. In particular, the methods and compositions are effective at interfering with the activity of host cell proteins required in viral replication. For example, an embodiment of the invention is directed to methods and compositions comprising RNA sequences to which the host cell proteins TIAR and/or TIA-1 bind.07-23-2009
20090062230NOVEL CATIONIC 17 ALPHA-SUBSTITUTED-ESTRADIOL DERIVATIVES USEFUL AS ANTI-CANCER AGENT - The present invention provides a novel series of cationic, lipid-based, 17α-substituted-estradiol derivatives. The present invention further provides a process for the preparation of a novel series of 17α-substituted-estradiol derivatives. The invention also provides information about highly selective anticancer activities of these molecules in estrogen responsive cell lines. The compound elicits high level of toxicity to gynecological cancer cell lines such as MCF-7, T47D (estrogen receptor positive cell lines), MDA-MB-468 (estrogen receptor knock-out cell line), Hela (cervical cancer). The present class of cationic lipid-based, estradiol derivatives is likely to find specific use in developing target specifically deliverable anticancer drugs for the treatment of gynecological cancers that are most prevalent in women population irrespective of ethnicity.03-05-2009
20090062225COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF A GENE FROM THE JC VIRUS - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a gene from the JC Virus. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by JC virus expression and the expression of a gene from the JC Virus using the pharmaceutical composition; and methods for inhibiting the expression of a gene from the JC Virus in a cell.03-05-2009
20090143319Transcription factor decoy - Double-stranded oligonucleotides useful as decoy oligonucleotides having a high binding ability to a transcription factor and having a reduced cytotoxicity are disclosed. Each of the double-stranded oligonucleotides is formed by hybridization of a sense strand oligonucleotide of the following Formula A:06-04-2009
20090143326MICROMIRs - The present invention relates to very short heavily modified oligonucleotides which target and inhibit microRNAs in vivo, and their use in medicaments and pharmaceutical compositions.06-04-2009
20090005337Pde11a Mutations in Adrenal Disease - The invention provides previously uncharacterized variants of PDE11A that are correlated with a newly discovered form of Cushing Syndrome that presents at a young age. The invention also provides methods useful to research, screen for, treat, or prevent diagnose the disease using the PDE11A variants, as well as other methods relating thereto.01-01-2009
20090005334Nucleic acid derivatives - A compound which comprises a backbone having a plurality of chiral carbon atoms, the backbone bearing a plurality of ligands each being individually bound to a chiral carbon atom of the plurality of chiral carbon atoms, the ligands including one or more pair(s) of adjacent ligands each containing a moiety selected from the group consisting of a naturally occurring nucleobase and a nucleobase binding group, wherein moieties of the one or more pair(s) are directly linked to one another via a linker chain; building blocks for synthesizing the compound; and uses of the compound, particularly in antisense therapy.01-01-2009
20080318894RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF HER3 - The invention relates to oligomer compounds (oligomers), which target HER3 mRNA in a cell, leading to reduced expression of HER3 and/or HER2 and/or EGFR. Reduction of HER3 and/or HER2 and/or EGFR expression is beneficial for a range of medical disorders, such hyperproliferative disorders (e.g., cancer). The invention provides therapeutic compositions comprising oligomers and methods for modulating the expression of HER3 and/or HER2 and/or EGFR using said oligomers, including methods of treatment.12-25-2008
20080318896Methods and Compositions for Controlling of Efficacy of RNA Silencing - Based at least in part on an understanding of the mechanisms by which small RNAs (e.g., naturally-occurring miRNAs) mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, the effect of the position of the mismatches, etc. Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing.12-25-2008
20080318889Antiproliferative activity of G-rich oligonucleotides and method of using same to bind to nucleolin - Compositions and methods for modulating tumor proliferation in an individual are provided. The methods employ nucleolin-binding agents, such as aptamers. The aptamers of the present invention can be used to modulate the proliferation of malignant, dysplastic, hyperproliferative, and/or metastatic cells through interference with molecular interactions and functions of nucleolin in the tumor cell.12-25-2008
20080318887Antiproliferative activity of G-rich oligonucleotides and method of using same to bind to nucleolin - Compositions and methods for modulating tumor proliferation in an individual are provided. The methods employ nucleolin-binding agents, such as aptamers. The aptamers of the present invention can be used to modulate the proliferation of malignant, dysplastic, hyperproliferative, and/or metastatic cells through interference with molecular interactions and functions of nucleolin in the tumor cell.12-25-2008
20080318890Antiproliferative activity of G-rich oligonucleotides and method of using same to bind to nucleolin - Compositions and methods for modulating tumor proliferation in an individual are provided. The methods employ nucleolin-binding agents, such as aptamers. The aptamers of the present invention can be used to modulate the proliferation of malignant, dysplastic, hyperproliferative, and/or metastatic cells through interference with molecular interactions and functions of nucleolin in the tumor cell.12-25-2008
20080318882Method of Delivery of Nucleic Acids to Peripheral Neurons - The invention provides for methods for delivering a nucleic acid into a peripheral neuron by identifying a target neuron in a dorsal root ganglion and intrathecally delivering a vector comprising the nucleic acid to the dorsal root ganglion neuron. The nucleic acid may encode a neurotrophic factor that may be used to treat a peripheral neuropathy or, in conjunction with a nerve guide conduit, to treat a transected peripheral nerve.12-25-2008
20080318885Method for Modulating Responsiveness to Steroids - The present invention makes it possible to enhance steroid efficacy in a steroid refractory patient afflicted with an inflammatory condition not responding or responding poorly or inadequately to anti-inflammatory treatment, by administering an effective amount of an oligonucleotide having the sequence 12-25-2008
20080234221TRANSFECTION AGENTS - The present invention provides compounds, compositions and methods that enhance the transfer of an agent into a cell. The agents can include polypeptides, polynucleotides such as genes and antisense nucleic acids, and other molecules. In some embodiments, the agents are modulating agents that can modulate a cellular activity or function when introduced into the cell. The compounds, compositions and methods are useful for introducing agents such as genes into individual cells, as well as cells that are present as a tissue or organ.09-25-2008
20080234219Compositions and Methods for Increasing Bone Mineralization - A novel class or family of TGF-β binding proteins is disclosed. Also disclosed are assays for selecting molecules for increasing bone mineralization and methods for utilizing such molecules.09-25-2008
20080234218Sirna For Inhibiting Il-6 Expression and Composition Containing Them - Disclosed is a double-strand siRNA capable of suppressing interleukin-6 (IL-6) expression, and a pharmaceutical composition containing the same. The siRNA of the present invention and the pharmaceutical composition containing the same may be useful to treat inflammatory diseases, autoimmune diseases, neoplasmic disease and central nervous system diseases, which are all associated with the over-expressed IL-6 level, by lowering an IL-6 level.09-25-2008
20080234216TGF-beta type receptor cDNAs encoded products and uses therefor - DNA encoding TGF-β type III receptor of mammalian origin, DNA encoding TGF-β type II receptor of mammalian origin, TGF-β type III receptor, TGF-β type II receptor and uses therefor.09-25-2008
20080234213Oncogenic regulatory RNAs for diagnostics and therapeutics - A method of identifying regulatory RNAs, including miRNAs, using insertional mutagenesis to generate tumors in mice and determining the human orthologs is disclosed. Further, specific miRNA sequences are identified. The causal nature and expression patterns of these regulatory RNAs and miRNAs in human tumors demonstrate their utility in diagnosis and therapy of cancer. Furthermore, a set of co-mutations that act in conjunction with miRNAs in tumor formation is disclosed.09-25-2008
2009024748250 Human Secreted Proteins - The present invention relates to novel human secreted proteins and isolated nucleic acids containing the coding regions of the genes encoding such proteins. Also provided are vectors, host cells, antibodies, and recombinant methods for producing human secreted proteins. The invention further relates to diagnostic and therapeutic methods useful for diagnosing and treating diseases, disorders, and/or conditions related to these novel human secreted proteins.10-01-2009
20090118215Bioresorbable Controlled-Release Composition - A novel method for the preparation of a highly densified and at least partly, preferably fully or almost fully hydrated ceramic for use in the preparation of a pharmaceutical composition notably for controlled-release of one or more therapeutically, prophylactically and/or diagnostically active substance. The method involves a concomitant step of hydrating and densifying a bioresorbable and hydratable ceramic such as calcium sulphate. The invention also relates to compositions comprising such a highly densified ceramic. The pharmaceutical composition is useful for targeted and controlled local prolonged release of active substances, e.g. anti-cancer agents, whereby the spectrum and severity of side effects are minimized due to an optimized local concentration-time profile.05-07-2009
20090023671Rnai Agents for Maintenance of Stem Cells - The present invention provides compositions and methods suitable for delivering RNAi agents against genetic targets in stem cells so as to direct cell growth and differentiation.01-22-2009
20090118213RNA ANTAGONIST COMPOUNDS FOR THE INHIBITION OF APO-B100 EXPRESSION - Oligonucleotides directed against the Apo-B100 gene are provided for modulating the expression of Apo-B100. The compositions comprise oligonucleotides, particularly antisense oligonucleotides, targeted to nucleic acids encoding the Apo-B100. Methods of using these compounds for modulation of Apo-B100 expression and for the treatment of diseases associated with either overexpression of Apo-B100, expression of mutated Apo-B100 or both are provided. Examples of diseases are cancer such as lung, breast, colon, prostate, pancreas, lung, liver, thyroid, kidney, brain, testes, stomach, intestine, bowel, spinal cord, sinuses, bladder, urinary tract or ovaries cancers. The oligonucleotides may be composed of deoxyribonucleosides or a nucleic acid analogue such as for example locked nucleic acid or a combination thereof.05-07-2009
20090023676RNA Interference Mediated Inhibition of MAP Kinase Gene Expression or Expression of Genes Involved in MAP Kinase Pathway Using Short Interfering Nucleic Acid (SiNA) - The present invention concerns methods and reagents useful in modulating MAP kinase gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against c-JUN, JNK, p38, and ERK gene expression, useful in the treatment of cancer, inflammation, obesity and insulin resistance (e.g. Type I and Type II diabetes).01-22-2009
20090023674snRNA gene-like transcriptional units and uses thereof - By a computer search for upstream promoter elements (DSE, PSE) typical of small nuclear RNA (snRNA) genes, we have identified a number of previously unrecognized, putative transcription units whose predicted products are novel noncoding RNAs with homology to protein-coding genes. By elucidating the function of one of them, we provide evidence for the existence of a sense/antisense-based gene regulation network where part of the Pol III transcriptome could control its Pol II counterpart.01-22-2009
20090023673LIPID CONTAINING FORMULATIONS - Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.01-22-2009
20090023670Regulation of Transgene Expression by RNA Interference - Expression of transgenes delivered into a host organism cells can be regulated by RNA effector molecules delivered to or present in the host organism cells. Regulation can be mediated by delivery of RNA interference inducing molecules that target the transgene mRNA or by incorporating engineered RNA effector binding sites into the transgene. Temporary or long term regulation of expression can be achieved depending on the nature and dosing of RNA effector.01-22-2009
20090082299CODON OPTIMIZED IL-15 AND IL-15R-ALPHA GENES FOR EXPRESSION IN MAMMALIAN CELLS - The present invention provides for nucleic acids improved for the expression of interleukin-15 (IL-15) in mammalian cells. The invention further provides for methods of expressing IL-15 in mammalian cells by transfecting the cell with a nucleic acid sequence comprising a codon optimized IL-15 sequence. The present invention further provides expression vectors, and IL-15 and IL 15 receptor alpha combinations (nucleic acid and protein) that increase IL-15 stability and potency in vitro and in vivo. The present methods are useful for the increased bioavailability and biological effects of IL-15 after DNA, RNA or protein administration in a subject (e.g. a mammal, a human).03-26-2009
20090054362COMPOUNDS - The present invention provides novel aptamer derivatives which are useful in binding to and neutralising viruses. Pharmaceutical formulations comprising the aptamers and the use of the aptamers in screening for useful compounds are also provided.02-26-2009
20090062228piRNA and uses related thereto - The invention relates to small single stranded RNAs and analogs thereof (collectively “piRNA” herein), compositions comprising such piRNAs, and their uses in regulating target gene expression or as markers for certain disease states.03-05-2009
20090062227METHODS AND COMPOSITIONS FOR INDUCING APOPTOSIS BY STIMULATING ER STRESS - The present invention provides a method for inducing apoptosis in selected cells by aggravating ER-stress. The aggravation of ER-stress is achieved in a specific manner by inhibiting SERCA (sarcoplasmic/endoplasmic reticulum calcium ATPase), leading to elevated level of cytoplasmic calcium concentration, yet without inhibiting the activity of COX-2 (cyclooxygenase-2) or triggering the release of histamine. Induction of apoptosis may be enhanced by first inducing or further aggravating ER-stress through inhibition of proteasome or proteases. Also provided are compounds and compositions useful as ER-stress aggravating agents, methods for screening, selecting, identifying and designing the same and methods for treating diseased conditions by inducing apoptosis through specific and selective aggravation of ER-stress.03-05-2009
20090062226Method and Compositions for Inhibiting MAGE Protein Interaction With KAP-1 - Method for inhibiting tumor cell formation or tumor cell growth, and method for inducing apoptosis in sperms, the method comprising administering to a patient in need thereof an antagonist that inhibits the binding of MAGE protein to KAP-1, thereby inhibiting MAGE gene function. Also disclosed are pharmaceutical compositions comprising the same, and method for screening a substance that inhibits MAGE protein binding to KAP-1.03-05-2009
20090062224Therapeutic use of cpg oligodeoxynucleotide for skin disease - Disclosed is the therapeutic use of CpG oligodeoxynucleotides for skin diseases. The CpG oligodeoxynucleotides (CpG ODNs) of the present invention show excellent immunoactive effects against skin diseases in both cases of CpG ODNs with a phosphorothioate backbone and CpG ODNs with a phosphodiester backbone.03-05-2009
20090062229METHOD AND COMPOSITION FOR REDUCING THE EXPRESSION OF ROCK-II - Methods and compositions for reducing the expression of Rho-associated, coiled-coil containing protein kinase 2 (ROCK-II) are provided.03-05-2009
20090054367MYCOBACTERIAL ANTIGENS EXPRESSED UNDER LOW OXYGEN TENSION - A method is provided for identifying mycobacterial genes that are induced or up-regulated under continuous culture conditions defined by a dissolved oxygen tension of up to 10% air saturation measured at 37° C. when compared with a dissolved oxygen tension of at least 40% air saturation measured at 37° C. Said induced or up-regulated genes form the basis of nucleic acid vaccines, or provide targets to allow preparation of attenuated mycobacteria for vaccines against mycobacterial infections. Similarly, peptides encoded by said induced or up-regulated genes are employed in vaccines. In a further embodiment, the identified genes/peptides provide the means for identifying the presence of a mycobacterial infection in a clinical sample by nucleic acid probe or antibody detection.02-26-2009
20090054366RNAi MEDIATED KNOCKDOWN OF NUMA FOR CANCER THERAPY - This invention relates to the use of short interfering nucleic acid molecules (siRNA) to inhibit Nuclear Mitotic Apparatus Protein (NuMA) gene expression and their use in treatment of disease, including cancer.02-26-2009
20090054363Genetic markers and methods for detecting and treating cancers - The present invention provides genetic markers, SOX5 and SPARC, for distant metastasis and poor prognosis of detection of the high risk potential for cancer patients. In addition, the present invention also provides a method to predict the risk potential for cancer patients with distant metastasis and poor prognosis. This method comprises obtaining a tissue sample from a patient, evaluating the expression levels of the SOX5 and/or SPARC genetic markers in the sample; and comparing the expression levels of genetic markers with those of non-cancerous tissues. The patient is determined to have the high risk of distant metastasis or poor prognosis when the expression level of SOX5 is higher, or when the expression level of SPARC is lower, than that of non-cancerous tissue. Furthermore, the identified genetic marker SOX5 and/or SPARC can also be used for cancer targeted therapy, because down regulation of SOX5 and/or up regulation of SPARC expression in NOD-SCID can retard tumor growth and inhibit cell proliferation, migration, invasion and metastasis.02-26-2009
20090054364Methods and compositions for delivery of pharmaceutical agents - Methods and compositions for delivering pharmaceutical agents into cells, in particular urothelial cells of the bladder, are provided. In the methods and compositions of the invention, a solubilized cholesterol composition is used to facilitate delivery of pharmaceutical agents. Preferably, the cholesterol is solubilized by a cyclodextrin (e.g., methyl-β-cyclodextrin) and the pharmaceutical agent comprises a polynucleotide and either a cationic lipid, a cationic polymer or a dendrimer. Improved methods for transfecting polynucleotides into cells thus are also provided, using cationic lipids, cationic polymers or dendrimers and solubilized cholesterol, wherein the transfection efficiency is enhanced compared to use of cationic lipids, cationic polymers or dendrimers alone. Preferred methods of the invention involve transfecting polynucleotides into urothelial cells, preferably for therapeutic treatment of bladder cancer using, for example, a polynucleotide(s) encoding an interleukin(s), an interferon(s), a colony stimulating factor(s) and/or a tumor suppressor(s).02-26-2009
20090099114Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.04-16-2009
20090082300MODULATION OF TRANSTHYRETIN EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of transthyretin. The compositions comprise oligonucleotides, targeted to nucleic acid encoding transthyretin. Methods of using these compounds for modulation of transthyretin expression and for diagnosis and treatment of diseases and conditions associated with expression of transthyretin are provided.03-26-2009
20090082294Diagnosis, prevention and treatment of cancer - Nucleic acid molecules useful in the treatment of cancer are provided. The nucleic acid molecules may include a short interfering ribonucleic acid molecule directed against fer mRNA. A kit for the treatment of cancer including a short interfering ribonucleic acid molecule of the presently described subject matter, as well as a pharmaceutical composition containing a short interfering ribonucleic acid molecule, is also provided. In addition, a method of treating cancer in an individual in which expression of fer in cells or tissues of an individual is inhibited using a short interfering ribonucleic acid molecule is provided.03-26-2009
20090082296MYCOBACTERIAL ANTIGENS EXPRESSED DURING LATENCY - A method is provided for identifying mycobacterial genes that are induced or up-regulated under culture conditions that are nutrient-starving and which maintain mycobacterial latency, said conditions being obtainable by batch fermentation of a mycobacterium for at least 20 days post-inoculation, when compared with culture conditions that are not nutrient-starving and which support exponential growth of said mycobacterium. Said induced or up-regulated genes form the basis of nucleic acid vaccines, or provide targets to allow preparation of attenuated mycobacteria for vaccines against mycobacterial infections. Similarly, peptides encoded by said induced or up-regulated genes are employed in vaccines. In a further embodiment, the identified genes/peptides provide the means for identifying the presence of a mycobacterial infection in a clinical sample by nucleic acid probe or antibody detection.03-26-2009
20090082295COMBINATIONS AND METHODS OF USING AN IMMUNOMODULATORY OLIGODEOXYNUCLEOTIDE - The present invention relates to combination therapies for the treatment of cancer. The combination of agents include oligonucleotides and one or more chemotherapeutic agents.03-26-2009
20090082291Methods of treatment of acute renal failure - The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a human p53 gene. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating a patient suffering from alopecia or acute renal failure or other diseases comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The alopecia may be induced by chemotherapy or radiotherapy, and the patient may be suffering from cancer, in particular breast cancer.03-26-2009
20090192114miR-10 Regulated Genes and Pathways as Targets for Therapeutic Intervention - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-10, using miR-10 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.07-30-2009
20090192109Compositions for diagnosis and therapy of diseases associated with aberrant expression of Kremen and/or Wnt - The present invention relates to a composition useful for the diagnosis and therapy of diseases associated with aberrant expression of the gene encoding the receptor Kremen 1 and/or Kremen 2 e.g. tumors or diseases of the kidneys, bones and eyes, lipid and glucose metabolism and obesity. The present invention also relates to a pharmaceutical composition containing a compound which is capable of modifying (a) the expression of the gene encoding Kremen 1 and/or Kremen 2 or (b) the activity of the Kremen 1 and/or Kremen 2 receptor.07-30-2009
20090082289Adenoviral vectors having a protein IX deletion - This invention provides a recombinant adenovirus expression vector characterized by the partial or total deletion of the adenoviral protein IX DNA and having a gene encoding a foreign protein or a functional fragment or mutant thereof. Transformed host cells and a method of producing recombinant proteins and gene therapy also are included within the scope of this invention.03-26-2009
20090105177MODULATION OF DIACYLGLYCEROL ACYLTRANSFERASE 1 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of diacylglycerol acyltransferase 1. The compositions comprise oligonucleotides, targeted to nucleic acid encoding diacylglycerol acyltransferase 1. Methods of using these compounds for modulation of diacylglycerol acyltransferase 1 expression and for diagnosis and treatment of disease associated with expression of diacylglycerol acyltransferase 1, such as obesity and obesity-related conditions, are provided.04-23-2009
20080318888Antiproliferative activity of g-rich oligonucleotides and method of using same to bind to nucleolin - Compositions and methods for modulating tumor proliferation in an individual are provided. The methods employ nucleolin-binding agents, such as aptamers. The aptamers of the present invention can be used to modulate the proliferation of malignant, dysplastic, hyperproliferative, and/or metastatic cells through interference with molecular interactions and functions of nucleolin in the tumor cell.12-25-2008
20090137505Method for Predicting and Identifying Target mRnas Controlled By Functional Rnas and Method of Using the Same - It is intended to identify or estimate miRNAs and one or more target genes (target mRNAs) targeted thereby.05-28-2009
20080261912Bispecific Antisense Oligonucleotides that Inhibit IGFBP-2 and IGFBP-5 and Methods of Using Same - Bispecific antisense oligonucleotides which consist essentially of a sequence of bases that is complementary to portions of both the gene encoding human IGFBP-2 and the gene encoding human IGFBP-5 are useful in as antisense therapeutics in the treatment of endocrine-regulated cancers.10-23-2008
20080261911Uses of diphenyl/diphenylamine carboxylic acids - The present invention demonstrates that chemical-induced degradation of Sp proteins by a specific sub-class of non-steroidal anti-inflammatory drugs inhibited cancer cell growth, angiogenesis and metastasis of cancer cells. The inhibitory effects of these compounds were demonstrated in vitro and in vivo. Hence, the results discussed herein indicate that these compounds can be used to inhibit cell growth, angiogenesis, and metastasis in cancers such as pancreatic cancer, esophageal cancer, breast cancer, prostate cancer, colon cancer, bladder cancer, and ovarian cancer.10-23-2008
20080261909Gene Delivery to Organs - Application of a virus with poloxamer alone onto atria results in diffuse epicardial gene transfer with negligible penetration into the myocardium. Progressive increases in protease concentration, however, allow transmural gene transfer. After protease exposure, echocardiographic left atrial diameter does not change. Left atrial ejection fraction decreases on post-operative day 3, but returns to baseline by day 7. At appropriate protease concentrations, tissue tensile strength is unaffected by the procedure. Transmural atrial gene transfer can be effected using this direct “painting” method.10-23-2008
20080261908MicroRNA-based methods and compositions for the diagnosis, prognosis and treatment of breast cancer - The present invention provides novel methods and compositions for the diagnosis, prognosis and treatment of breast cancer. The invention also provides methods of identifying anti-breast cancer agents.10-23-2008
20080261910Diagnostic Methods Based on Polymorphisms of Glucosyltransferase-Like Protein - The present invention arises from the identification of an association between the gene encoding glucosyltransferase-like protein (GT) and osteoarthritis (OA). It therefore relates to diagnostic techniques for determining a patient's susceptibility to develop OA by detecting all or part of the GT gene, its precursors or products (mRNA, cDNA, genomic DNA, or protein). In particular, the invention relates to methods and materials for analysing allelic variation in the GT gene, and to the use of GT polymorphisms in the identification of an individuals' risk to develop OA. The invention is also directed to methods for identifying modulators of OA, which modulate the GT gene or its encoded protein.10-23-2008
20080261907COMPOSITIONS AND METHODS OF SPHINGOSINE KINASE INHIBITORS IN RADIATION THERAPY OF VARIOUS CANCERS - The present invention relates to Sphingosine kinase inhibitors that are useful for treating various cancers. The invention further relates to compositions and methods of SPK inhibitors, including siRNAs, which specifically block gene expression of SPK and potentiates the effect of radiation in the treatment of various cancers.10-23-2008
20080261906METHODS AND COMPOSITIONS FOR IDENTIFYING ANTI-HCV AGENTS - The invention features methods and compositions for screening for agents that modulate replication of a virus, particularly Flaviviridae virus (particularly hepatitis C virus (HCV)), where the methods provide for detection of agents that modulate the binding of TBC and NS5A, the inhibition of TBC activity, inhibition of Rab1 activity, and/or the expression of the TBC protein and/or Rab1 protein. The invention also features methods of controlling viral replication, and agents useful in such methods.10-23-2008
20080261905Modified Nucleosides for Rna Interference - The present invention relates to the use of modified nucleotides and single or double stranded oligonucleotides having at least one of said modified nucleotides for performing RNA interference. The modified nucleotides are selected from 6-membered ring containing nucleotides such as hexitol, altritol, O-substituted or O-alkylated altritol, cyclohexenyl, ribo-cyclohexenyl and O-substituted or O-alkylated ribo-cyclohexenyl nucleotides. The present invention also relates to novel modified nucleosides or nucleotides and to the use of the novel modified nucleosides and nucleotides in single or double stranded oligonucleotides for RNA interference, antisense therapy or other applications.10-23-2008
20080261904Chimeric Gapped Oligomeric Compounds - The present invention provides double stranded compositions wherein the first strand comprises three different regions and the second strand is native RNA. Each region has native or modified ribofuranosyl sugar moieties that are different than those of the other two regions. At least a portion of the first oligomeric compound is complementary to and hybridizes to a nucleic acid target. The present invention also provides methods for modulating gene expression using the modified oligomeric compounds and compositions of oligomeric compounds.10-23-2008
20090105183Pharmaceutical composition containing decoy and method of using the same - A pharmaceutical composition for performing treatment against a skin disease, the pharmaceutical composition comprising at least one decoy and a pharmaceutically acceptable carrier. The at least one decoy may be selected from the group consisting of an NF-κB decoy, a STAT-1 decoy, a GATA-3 decoy, a STAT-6 decoy, an AP-1 decoy and an Ets decoy. The at least one decoy may be an oligonucleotide including at least two decoys bonded to each other, the at least two decoys being selected from the group consisting of an NF-κB decoy, a STAT-1 decoy, a GATA-3 decoy, a STAT-6 decoy, an AP-1 decoy and an Ets decoy. The skin disease may be atopic dermatitis, psoriasis vulgaris, contact dermatitis, keloid, bedsore, ulcerative colitis, or Crohn's disease.04-23-2009
20090105182RNAi-MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR 1-RELATED TARGETS FOR TREATMENT OF NEOVASCULARIZATION-RELATED CONDITIONS - RNA interference is provided for inhibition of stromal cell-derived factor 1 (SDF1)-related targets in pathologic neovascularization-related conditions, including those cellular changes resulting from the signal transduction activity of the SDF1 targets that lead directly or indirectly to ocular neovascularization, abnormal angiogenesis, retinal vascular permeability, retinal edema, diabetic retinopathy particularly proliferative diabetic retinopathy, diabetic macular edema, exudative age-related macular degeneration, sequela associated with retinal ischemia, and posterior segment neovascularization, for example.04-23-2009
20090105179DRUG CARRIERS - Compositions that can include a cationic polymeric carrier, targeting agent, and therapeutic agent are disclosed herein. The therapeutic agent may have a therapeutic activity such as inhibiting fibrosis within a target organ or tissue or inhibiting the growth of a cancer cell.04-23-2009
20090105178RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET-DERIVED ENDOTHELIAL CELL GROWTH FACTOR (ECGF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating platelet-derived endothelial cell growth factor and/or receptor (ECGF1 and/or ECGF1r) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of ECGF1 and/or ECGF1r gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of ECGF1 and/or ECGF1r genes.04-23-2009
20090105173Prevention and treatment of acute renal failure and other kidney diseases by inhibition of p53 by siRNA - The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a human p53 gene. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating a patient suffering from acute renal failure or other kidney diseases comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient.04-23-2009
20090105180Ex vivo and in vivo expression of the thrombomodulin gene for the treatment of cardiovascular and peripheral vascular diseases - The present invention relates to methods and compositions for treatment of cardiovascular and peripheral vascular diseases using ex vivo and in vivo gene delivery technologies. One aspect of the present invention relates to a method for treating a vascular disease by introducing a DNA sequence encoding a TM protein or its variant into a segment of a blood vessel ex vivo using a gutless adenovirus vector. Another aspect of the present invention is to provide a gutless adenovirus vector carrying a transgene, such as a gene encoding TM protein or its variant.04-23-2009
20090105176Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.04-23-2009
20090105172Stabilized Aptamers to PSMA and Their Use as Prostate Cancer Therapeutics - The present invention provides stabilized, high affinity nucleic acid ligands to PSMA. Methods for the identification and preparation of novel, stable, high affinity ligands to PSMA using the SELEX™ method with 2′-O-methyl substituted nucleic acids, and cell surface SELEX™ are described herein. Also included are methods and compositions for the treatment and diagnosis of disease characterized by PSMA expression, using the described nucleic acid ligands.04-23-2009
20090105174NUCLEIC ACIDS HYBRIDIZABLE TO MICRO RNA AND PRECURSORS THEREOF - Methods and compositions relating to nucleic acids targeting certain miRNA molecules are disclosed. The nucleic acids are useful in methods of increasing nuclear concentration of FKHR protein, decreasing cell viability, and treating cancer.04-23-2009
20090105171Methods for Regenerating and Repairing Damaged Tissues Using Adrenomedullin - [Problems to be Solved] An objective of the present invention is to provide methods for regenerating or repairing damaged tissues using adrenomedullin. Another objective is to provide pharmaceutical agents that comprise adrenomedullin as an active ingredient for regenerating or repairing damaged tissues.04-23-2009
20090105170Multiple Heat Shock Elements - A DNA molecule is provided which comprises at least 2 consensus sequences, each consensus sequence consisting of 3 pentameric units, said pentameric units having a sequence XGAAY or an inverse sequence Y′TTCX′, X being selected from the group consisting of A, T, G, and C, and Y of at least one, preferably two, still preferred all three, of said 3 pentameric units of at least one consensus sequence being selected from the group consisting of A, T, and C, the Y of the remaining pentameric units of said at least one consensus sequence being selected from the group consisting of A, T, G, and C, whereby in the case that said DNA molecule comprises more than 6 consensus sequences, Y of all pentameric units is selected from the group consisting of A, T, G, and C.04-23-2009
20090105169ALLELE-SPECIFIC SILENCING OF DISEASE GENES - The present invention is directed to small interfering RNA molecules (siRNA) targeted against an allele of interest, and methods of using these siRNA molecules.04-23-2009
20080318886Methods of Increasing Cancer Sensitivity to Chemotherapeutic Agents Using Chimeric ISF35 - A method of treating cancer by administering ISF35 or analogous constructs. The treatment can allow an increased sensitivity to treatment with a chemotherapeutic agent.12-25-2008
20090099119RNA INTERFERENCE MEDIATED INHIBITION OF RAS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating RAS, e.g. K-RAS, H-RAS, and/or N-RAS gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of RAS, e.g. K-RAS, H-RAS, and/or N-RAS gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of RAS genes, such as K-RAS, H-RAS, and/or N-RAS.04-16-2009
20090099112Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.04-16-2009
20090209478COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE HAMP GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the HAMP gene (HAMP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the HAMP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HAMP gene expression and the expression of the HAMP gene using the pharmaceutical composition.08-20-2009
20080318884Methods for Altering Gene Expression and Methods of Treatment Utilizing Same - The present disclosure describes methods for altering the expression of a target gene comprising a rare cluster of codons, including, but not limited to, trinucleotide repeats. The method utilizes, in part, on amino acid deprivation or the limiting of specific charged tRNAs. The methods for altering target gene expression may be used in treatment methods to treat diseases in a subject organism in need of such treatment. Such methods for altering target gene expression have not been heretofore recognized in the art. Exemplary diseases that may be treated using the methods of the present disclosure include any disease where altering the expression of the target gene would provide treatment. Such diseases include all forms of cancer, ageing, infectious disease, metabolic disorders, inflammation, neurological disorders, diabetes, psychiatric disorders and diseases associated with trinucleotide repeats.12-25-2008
20090093435RNA INTERFERENCE MEDIATED INHIBITION OF GRB2 ASSOCIATED BINDING PROTEIN (GAB2) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating associated binding protein (GAB2) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of associated binding protein (GAB2) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of GAB2 genes. The small nucleic acid molecules are useful in the treatment of cancer, malignant blood disease (leukemia), inflammatory diseases or conditions, allergic diseases or conditions, or proliferative diseases or conditions.04-09-2009
20090093433SENSE mRNA THERAPY - The present invention describes novel methods for the stabilization of mRNA. These alterations increase stability of mRNA and enable its use in sense RNA therapy to transiantly express proteins in a cell. Accordingly, the present invention is directed to methods for making such modifications, compositions comprising such modifications, and the use of such compositions in treating disease states.04-09-2009
20090093432TUMOR INHIBITION BY MODULATING SPROUTY EXPRESSION OR ACTIVITY - Methods are provided for identifying compounds that decrease the expression or activity of an overexpressed Sprouty protein in certain cancers. Such compounds can be useful for treating cancers in which a Sprouty protein is overexpressed. Also provided are therapeutic formulations and pharmaceutical formulations for treating cancers characterized by overexpression of a Sprouty protein.04-09-2009
20090093428Nucleotide vector, composition containing such vector, and vaccine for immunization against hepatitis - Nucleotide vector composition containing such vector and vaccine for immunization against hepatitis. Nucleotide vector comprising at least one gene or one complementary DNA coding for at least a portion of a virus, and a promoter providing for the expression of such gene in muscle cells. The gene may be the S gene of the hepatitis B virus. A nucleotide vector composition when administered to even chronic HBV carriers is capable of breaking T cell tolerance to the surface antigens of hepatitis B virus. A vaccine preparation containing said bare DNA is injected into the host previously treated with a substance capable of inducing a coagulating necrosis of the muscle fibers.04-09-2009
20090082302MODULATION OF EIF4E-BP2 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of eIF4E-BP2. The compositions comprise oligonucleotides, targeted to nucleic acid encoding eIF4E-BP2. Methods of using these compounds for modulation of eIF4E-BP2 expression and for diagnosis and treatment of diseases and conditions associated with expression of eIF4E-BP2 are provided.03-26-2009
20090048196Membrane receptor for retinol binding protein mediates cellular uptake of vitamin A, methods of use and compositions - Provided is a method of diagnosing a condition associated with abnormal uptake of a retinoid comprising determining the amount of STRA6 expressed by a suspect cell and comparing the amount of expressed STRA6 in the suspect cell with the amount of expressed STRA6 in a normal cell. Also provided is a method for treating a condition associated with excessive uptake or insufficient uptake of a retinoid comprising administering to a subject in need thereof an amount of a drug effective for lowering or increasing expression or activity of STRA6 in cells. Also provided is a method of screening for a compound which alters the uptake of a retinoid by a cell comprising exposing the cell to the compound and determining if the expression or activity of STRA6 is altered.02-19-2009
20090048191Therapeutic molecules for modulating stability of vegf - This present invention discloses nucleic acid compositions and methods that are useful for treating ischemic conditions in animals, particularly in mammals such as humans. Specifically, the invention discloses nucleic acid molecules comprising or encoding a sequence that modulates the stability of a transcript from a vascular endothelial growth factor gene, as well as pharmaceutical compositions containing such molecules, which arm useful for modulating angiogenesis or vascularization, especially in methods for treating ischemic conditions.02-19-2009
20090048193Administration of the REG1 anticoagulation system - An improved method of administration of an aptamer and antidote system to regulate blood coagulation in a host is provided based on weight adjusted or body mass index-adjusted dosing of the components of the system to provide a desired pharmacodynamic response. In addition, a method of reversing activity of the aptamer to a desired extent is provided where an antidote dose is based solely on its relationship to the aptamer dose.02-19-2009
20090048201USE OF TARGETED CROSS-LINKED NANOPARTICLES FOR IN VIVO GENE DELIVERY - The in vivo delivery of nucleic acids is targeted by delivery of the nucleic acid in a complex with cross-linked nanoparticles; where the nanoparticles comprise cross-linked neutral amphipathic molecules, cationic amphipathic molecules and targeting amphipathic molecules. Optionally the cationic and targeting amphipathic molecules are also cross-linked. A targeting moiety present on the targeting amphipathic molecule provides for selective delivery of the complex to a predetermined target site, e.g. blood vessels, endothelial cells, tumor cells, liver cells, and the like.02-19-2009
20090048192Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation - The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having an alternating motif and another strand having a hemimer motif, a blockmer motif, a fully modified motif or a positionally modified motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.02-19-2009
20090105181Modulation of sodium channels by nicotinamide adenine dinucleotide - The present invention relates to the use of oxidized nicotinamide adenine dinucleotide (NAD04-23-2009
20080214489Aptamer-mediated intracellular delivery of oligonucleotides - Compositions and methods to mediate the intracellular and sub-cellular delivery of therapeutic, diagnostic and imaging agents, such as oligonucleotides. The compositions of the invention include nucleic acid conjugates comprising a delivery aptamer that is connected to an oligonucleotide. The delivery aptamer may be connected directly to the oligonucleotide, or may be connected indirectly to the oligonucleotide, such as through a linker. The oligonucleotide may be either a therapeutic, diagnostic or imaging oligonucleotide. The methods of the invention are used to increase potency, or alter distribution, half-life, metabolic fate, toxicity and other characteristics.09-04-2008
20080214487Methods and compositions for treatment of inflammatory disease using cadherin-11 modulating agents - A method for treating inflammatory joint diseases by inhibiting cadherin-11 mediated cellular function using a cadherin-11 modulating agent is provided. Also provided are screening assays for identifying pharmaceutical lead compounds capable of modulating cellular functions of cadherin-11 such as cell proliferation, apoptosis, factor secretion, and binding of cadherin-11 to cadherin-11 counter-receptor inhibiting binding of cadherin-11 to its counter-receptor either in the context of a cell or in soluble form.09-04-2008
20080214482Retromer-based assays and methods for treating alzheimer's disease - This invention provides a method for determining whether an agent causes an increase in the expression of a retromer complex protein. This invention further provides a method for determining whether an agent causes an increase in the activity of a retromer complex. This invention also provides a method for increasing the expression of a retromer complex protein in a cell. This invention provides a method for treating a subject afflicted with Alzheimer's disease. This invention further provides a pharmaceutical composition as well as an article of manufacture.09-04-2008
20090069266METHODS AND COMPOSITIONS FOR NUCLEIC ACID TRANSFER INTO CELLS - The present invention provides compositions and methods for increasing the transfer of nucleic acids into cells. In particular, the present invention provides for the use of inhibitors of HDAC6, a cytoplasmic histone deacetylase present in mammalian cells by, for example, small molecules or siRNA treatment, in increasing gene transfer and/or expression in cells in vitro and in vivo for research and gene therapy applications.03-12-2009
20090012031EZH2 Cancer Markers - The present invention relates to therapeutic targets for cancer. In particular, the present invention relates to small molecules and nucleic acids that target EZH2 expression in prostate cancer.01-08-2009
20090042830NOVEL ONCOGENE, RECOMBINANT PROTEIN DERIVED THEREFROM, AND USES THEREOF - The present invention identifies the total nucleotide sequence of a novel oncogene from human, which is directly involved in such a cancerization mechanism as for cervical cancer induced by HPV infection of cervical epithelial cell and the amino acid sequence of an oncogenic protein encoded thereby, and to provide a full-length polynucleotide encoding a peptide chain of the oncogenic protein derived from the novel oncogene, which can be used for recombinant production of the oncogenic protein, and the peptide chain of the oncogenic protein produced recombinantly therewith. Specifically, the present invention provides a novel oncogene polynucleotide from human involving development of cervical cancer, comprising a nucleotide sequence encoding an amino acid sequence of SEQ. ID. No. 1, particularly a polynucleotide of the nucleotide sequence of SEQ. ID. No. 2.02-12-2009
20090042829Nucleic Acid-Lipopolymer Compositions - Compositions, methods, and applications that increase the efficiency of nucleic acid transfection are provided. In one aspect, a pharmaceutical composition may include at least about 0.5 mg/ml concentration of a nucleic acid condensed with a cationic lipopolymer suspended in an isotonic solution, where the cationic lipopolymer includes a cationic polymer backbone having cholesterol and polyethylene glycol covalently attached thereto, and wherein the molar ratio of cholesterol to cationic polymer backbone is within a range of from about 0.1 to about 10, and the molar ratio of polyethylene glycol to cationic polymer backbone is within a range of from about 0.1 to about 10. The composition further may include a filler excipient.02-12-2009
20080318893Blockade of Calcium Channels - Knock-down of L-type calcium channel beta subunit (LTCCβ) attenuates the hypertrophic response both in vitro and in vivo without compromising systolic performance. Knock-down can be accomplished by administration of a vector encoding a short hairpin RNA which specifically modulates expression of LTCCβ. Suppression of the LTCCβ expression represents a therapeutic modality for cardiac hypertrophy.12-25-2008
20090075929IG20 SPLICE VARIANTS THERAPEUTICS FOR CANCER - Methods and compositions inhibit the growth of cancer cells by selectively down-regulating the expression of an IG20 splice variant including MADD. Specific knock-down of MADD splice variant resulted in the apoptosis of cancer cells. Interfering RNAs including small hairpin RNAs (shRNA) to down-regulate MADD expression in vivo are disclosed. Inhibition of MADD phosphorylation by Akt results in activation of cancer cell death. Down-regulation of MADD expression results in switching to apoptotic mode due to lack of MAPK activation upon TNF-α-based induction.03-19-2009
20090075926METHOD FOR IDENTIFYING AND MANIPULATING CELLS - The present application discloses a method of isolating or selecting stem cells from a mixed population containing stem cells, which includes the population of cells with a ligand specific for a truncated MUC1 receptor, wherein the presence of the truncated MUC1 receptor on the cells indicates that they are stem cells.03-19-2009
20090075925Methods and Compositions Related to APOBEC-1 Expression - Disclosed are methods and compositions related to ACF and to APOBEC-1.03-19-2009
20090143325RNA INTERFERENCE MEDIATED INHIBITION OF INTERLEUKIN AND INTERLEUKIN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of interleukin and/or interleukin receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of interleukin and/or interleukin receptor genes, IL-1, IL-2, IL-3, IL-4, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL-11, IL-12, IL-13, IL-14, IL-15, IL-16, IL-17, IL-18, IL-19, IL-20, IL-21, IL-22, IL-23, IL-24, IL-25, IL-26, and IL-27 genes and IL-1R, IL-2R, IL-3R, IL-4R, IL-5R, IL-6R, IL-7R, IL-8R, IL-9R, IL-10R, IL-11R, IL-12R, IL-13R, IL-14R, IL-15R, IL-16R, IL-17R, IL-18R, IL-19R, IL-20R, IL-21R, IL-22R, IL-23R, IL-24R, IL-25R, IL-26R, and IL-27R.06-04-2009
20090186845INTERFERING RNA MOLECULES - The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.07-23-2009
20090137517SENSITIZING A CELL TO CANCER TREATMENT BY MODULATING THE ACTIVITY OF A NUCLEIC ACID ENCODING RPS27L PROTEIN - The present invention refers to a method of sensitizing a cell to cancer treatment comprising modulating the activity of a nucleic acid which encodes the RPS27L protein by use of an oligonucleotide which is a RNAi agent or an antisense nucleotide molecule. The oligonucleotides of the present invention can be used in combination with at least one cytostatic drug for, e.g., chemotherapy. The present invention further refers to an expression vector comprising an oligonucleotide used in the method of the present invention as well as to a pharmaceutical comprising the oligonucleotides together with at least one chemotherapeutic agent used in the method of the present invention.05-28-2009
20090163432Therapeutic Agent for Corneal Diseases - The present invention relates to a treatment agent for a disease or a disorder caused by a reduction in corneal endothelial cells, comprising as an active component at least one nucleic acid molecule inhibiting the expression of a connexin 43 gene.06-25-2009
20090111766RAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES - Disclosed are recombinant adeno-associated viral (rAAV) vector compositions that are expressed in selected mammalian cells, such as pancreatic islets cells, and that encode one or more mammalian serpin or cytokine polypeptides having therapeutic efficacy in the amelioration, treatment and/or prevention of interleukin deficiencies, such as for example diabetes, and related diseases of the pancreas. Also disclosed are methods and compositions for preventing diabetes in a mammal, reducing the rate of disease progression, and ameliorating the symptoms of diabetes in humans at risk for developing such conditions.04-30-2009
20090111763LOADABLE POLYMERIC PARTICLES FOR BONE AUGMENTATION AND METHODS OF PREPARING AND USING THE SAME - Particles are provided for use in therapeutic and/or diagnostic procedures. The particles include poly[bis(trifluoroethoxy)phosphazene] and/or a derivatives thereof which may be present throughout the particles or within an outer coating of the particles. The particles may also include a core having a hydrogel formed from an acrylic-based polymer. Such particles may be provided for placement within defects in bone within the body of a mammal to augment structural support and facilitate osteogenesis without causing adverse reactions therein. The hydrogel core may further be used as a delivery vehicle for therapeutic agents to treat or retard pathologic processes within the bone defect during healing.04-30-2009
20090018098TARGETING THE ABSENCE: HOMOZYGOUS DNA DELETIONS AS SIGNPOSTS FOR CANCER THERAPY - The present application relates to methods and compositions for targeting a homozygous DNA deletion (HD) in cells. Methods, structures, vectors and compositions for activating a payload in cells having an HD are provided. In some embodiments, the method comprises administering, to a population of cells comprising at least one cell having an HD, a nucleic acid vector encoding two complementary protein fusion molecules and a payload such that the payload is selectively activated only in cells having a HD. HDs are attractive “negative” targets for cancer therapy because of their immutability and prevalence in cancer cells. Thus, in some embodiments methods of treating cancer by specifically delivering a payload, such as a toxin, to cancer cells comprising one or more particular HDs are provided.01-15-2009
20080269150Intra-vascular kidney gene therapy with plasmid encoding BMP-7 - The present invention relates to recombinant vectors expressing the BMP-7 polypeptide in host cells and to pharmaceutical compositions comprising such recombinant vectors. The invention also encompasses methods for prevention and/or treatment of both acute and chronic renal failure in mammals, advantageously in humans, dogs and cats, by intra-vascular kidney administration of the recombinant vectors and pharmaceutical compositions of the invention.10-30-2008
20090111767MODULATION OF INSULIN LIKE GROWTH FACTOR I RECEPTOR EXPRESSION - The present invention provides compositions and methods for modulating the expression of growth factor gene. In particular, this invention relates to compounds, particularly oligonucleotide compounds, which, in preferred embodiments, hybridize with nucleic acid molecules encoding the Insulin Like Growth Factor I receptor (IGF-I receptor or IGF-IR) and in particular human IGF-IR. Such compounds are exemplified herein to modulate proliferation which is relevant to the treatment of proliferative and inflammatory skin disorders and cancer. It will be understood, however, that the compounds can be used for any other condition in which the IGF-IR is involved including inflammatory conditions.04-30-2009
20090111768Regulators of protein misfolding and neuroprotection and methods of use - Polynucleotide molecules and the proteins encoded by the molecules, diagnostic and treatment methods for neurological disorders characterized by protein aggregation are provided. Genes are described herein that affect the misfolding of, and subsequent aggregation of, aggregation-prone proteins such as alpha-synuclein and have implications for the diagnosis and treatment of neurological diseases related to protein aggregation such as Parkinson's disease. Knockdown of expression of the genes described herein using RNAi results in alpha-synuclein protein aggregation in a 04-30-2009
20090111762METHOD OF DIAGNOSING AND TREATING CANCER USING B-CATENIN SPLICE VARIANTS - The invention relates to method of diagnosing and treating cancer, in particular β-catenin related cancers. The invention further relates to methods of identifying CTNNB1 related cancer CTNNB1 therapeutics.04-30-2009
20090048194Vagal Afferent Neurons as Targets for Treatment - A method of identifying a compound capable of reducing or preventing prolonged sensory neuron hyper-excitability comprising the steps of: (a) administering the compound to an experimental non-human animal having prolonged sensory neuron hyper-excitability; (b) generating an expression profile of the genes modulated in the Nodose Ganglia (NG) of the animal of step (a); (c) comparing the expression profile obtained in (b) with the expression profile of a corresponding panel of genes expressed in the NG of an experimental non-human animal having no prolonged sensory neuron hyper-excitability; wherein a positive correlation of the expression profiles is indicative that the compound is capable of reducing or preventing prolonged sensory neuron hyper-excitability in NG.02-19-2009
20090069265siRNA targeting inner centromere protein antigens (INCENP) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for INCENP.03-12-2009
200900692634'-THIOARABINONUCLEOTIDE-CONTAINING OLIGONUCLEOTIDES, COMPOUNDS AND METHODS FOR THEIR PREPARATION AND USES THEREOF - Oligonucleotides comprising one or more 4′-thioarabinonucleotides are described, as well as uses thereof for applications such as antisense- and RNAi-based gene silencing. 4′-thioarabinose-based phosphoramidite and H-phosphonate compounds are also described, as well as uses thereof for the synthesis of oligonucleotides comprising one or more 4′-thioarabinonucleotides.03-12-2009
20090069257Method of achieving persistent Transgene expression - Non-inflammatory vector compositions are provided that are suitable for repeated transgene delivery and that result in persistent transgene expression. The compositions are non-inflammatory, the present compositions are suitable for readministration and do not induce expression-limiting immune or inflammatory responses. Thus, these compositions are useful in methods of repeated administration to achieve persistent transgene expression, and are especially suited to treating genetic, acquired and inflammation-associated conditions.03-12-2009
20090069258RECOMBINANT ANTIBODIES SPECIFIC FOR BETA-AMYLOID ENDS, DNA ENCODING AND METHODS OF USE THEREOF - DNA encoding a recombinant antibody molecule end-specific for an amyloid-beta peptide, pharmaceutical compositions thereof, and a method for preventing or inhibiting progression of Alzheimer's Disease by introducing such a DNA molecule into brain cells to express the recombinant antibody molecule and prevent the accumulation of amyloid-beta peptides in the cerebrospinal fluid.03-12-2009
20090036396RNAi-RELATED INHIBITION OF TNFalpha SIGNALING PATHWAY FOR TREATMENT OF OCULAR ANGIOGENESIS - RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enzyme (TACE/ADAM17) mRNA expression. Silencing such TNFα targets, in particular, is useful for treating patients having a TNFα-related condition or at risk of developing a TNFα-related condition, such as ocular angiogenesis, retinal ischemia, and diabetic retinopathy.02-05-2009
20090036398Human G-Protein Coupled Receptor (HETGQ23) - Human G-protein coupled receptor polypeptides and DNA (RNA) encoding such polypeptides and a procedure for producing such polypeptides by recombinant techniques is disclosed. Also disclosed were methods for utilizing such polypeptides for identifying antagonists and agonists to such polypeptides and methods of using the agonists and antagonists therapeutically to treat conditions related to the underexpression and overexpression of the G-protein coupled receptor polypeptides, respectively. Also disclosed are diagnostic methods for detecting a mutation in the G-protein coupled receptor nucleic acid sequences and an altered level of the soluble form of the receptors.02-05-2009
20090036397RNAi-RELATED INHIBITION OF TNFa SIGNALING PATHWAY FOR TREATMENT OF MACULAR EDEMA - RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enzyme (TACE/ADAM17) mRNA expression. Silencing such TNFα targets, in particular, is useful for treating patients having or at risk of developing macular edema.02-05-2009
20080249055Use of Seh Inhibitors as Analgesics - The present invention provides methods and compositions for relieving pain and itching, of promoting wound healing, of reducing sickness behavior and of reducing inflammatory bowel disease or acne lesions in a subject by the topical administration of an inhibitor of soluble epoxide hydrolase, or of a cis-epoxyeicosatrienoic acid (“EET”), or by both.10-09-2008
20090137504MICRORNA TARGET SITE BLOCKING OLIGOS AND USES THEREOF - The present invention related to nucleic acids designed to prevent the binding of endogenous or exogenous microRNA and diagnostic and therapeutic uses thereof.05-28-2009
20090137511RNA INTERFERENCE MEDIATED INHIBITION OF PLACENTAL GROWTH FACTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating placental growth factor (e.g., PGF-1 or PlGF-1, PGF-2 or PlGF-2, and/or PGF-3 or PlGF-3) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of placental growth factor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of placental growth factor genes.05-28-2009
20090239816Multitargeting Interfering RNAs And Methods Of Their Use And Design - Interfering RNA molecules are now designed and produced with specificity for multiple binding sequences present in distinct genetic contexts in one or more pre-selected target RNA molecules and are used to modulate expression of the target sequences. Such a multitargeting interfering RNA approach provides a powerful tool for gene regulation.09-24-2009
20090048199Hibernation-Related Genes and Proteins, Activators and Inhibitors Thereof and Methods of Use - The present invention concerns methods and compositions for identifying genes and/or proteins involved in hibernation, activators and/or inhibitors of such genes or proteins, and methods of therapeutic use of such activators and/or inhibitors for treatment of a wide variety of diseases and/or medical conditions. In particular embodiments, such hibernation-related genes may include, but are not limited to, Adfp, Atr4, Cact, Myl6, Ca3, Ckm, Rps2, Lgmn, Fabpa, Fabph and Cyp51a1. Compounds that regulate the activities or functions of the Adfp, Atf4, Cact, Myl6, Ca3, Ckm, Rps2, Lgmn, Fabpa, Fabph and Cyp51a1 genes are known in the art and may be used for therapeutic treatment of diseases involving cardiovascular, gastrointestinal, respiratory, neurologic or immunologic function.02-19-2009
20090069259Treatment of neuropathic pain with zinc finger proteins - A variety of zinc finger proteins (ZFPs) and methods utilizing such proteins are provided for use in treating neuropathic pain. ZFPs that bind to a target site in genes that are aberrantly expressed in subjects having neuropathic pain are described. In addition, ZFPs that bind to a target site in genes expressed at normal levels in subjects experiencing neuropathic pain, modulation of whose expression results in decreased pain perception, are also provided. For example, genes that are over-expressed in the dorsal root ganglia (DRG) of pain patients (e.g., VR1, TRKA and/or Nav1.8) can be repressed, whereas genes that are under-expressed in the same populations can be activated.03-12-2009
20090093438RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating XIAP gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of XIAP gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of XIAP genes.04-09-2009
20090054361Pot1 alternative splicing variants - The present invention provides methods an compositions for diagnosis and treatment of carcinomas with aberrant expression patterns of POT 1. The invention also provides methods of identifying compounds that may modulate the cellular expression of POT 1. The invention further provides methods for treating subjects suffering from or at risk of developing a colorectal carcinoma.02-26-2009
20090215711Novel compositions and methods in cancer - The present invention relates to novel sequences for use in detection, diagnosis and treatment of cancers, especially lymphomas. The invention provides cancer-associated (CA) polynucleotide sequences whose expression is associated with cancer. The present invention provides CA polypeptides associated with cancer that present novel therapeutic targets against cancer. The present invention further provides diagnostic compositions and methods for the detection of cancer. The present invention provides monoclonal and polyclonal antibodies specific for the CA polypeptides. The present invention also provides diagnostic tools and therapeutic compositions and methods for screening, prevention and treatment of cancer.08-27-2009
20090209479THIOCARBON-PROTECTING GROUPS FOR RNA SYNTHESIS - Aspects of the invention include 2′ protected nucleoside monomers that are protected at the 2′ site with thiocarbon protecting groups. Thiocarbon protecting groups of interest include thiocarbonate, thionocarbonate, dithiocarbonate groups, as well as thionocarbamate protecting groups. Aspects of the invention further include nucleic acids that include the protecting groups of the invention, as well as methods of synthesizing nucleic acids using the protecting groups of the invention.08-20-2009
20080249050Compounds and methods to enhance rAAV transduction - Agents and methods to alter rAAV transduction are provided.10-09-2008
20080249052Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma - The present invention provides novel dystrophin mini/micro-genes that retain the essential biological functions of a full-length dystrophin gene. More particularly, the present invention provides to a series of synthetic mini/micro-dystrophin genes capable of restoring neuronal nitric oxide synthase (nNOS) to the sarcolemma. A method as well as a pharmaceutical composition for treatment of Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and X-linked Dilated Cardiomyopathy (XLDC) are also provided.10-09-2008
20080249054METHOD OF MEASURING LIPID DROPLETS AND APPLICATIONS OF USING THE SAME - The invention relates to methods of screening agents for the ability to regulate lipid metabolism and using said agents to treat diseases or disorders related to lipid metabolism (e.g., obesity, diabetes [non-insulin and insulin dependent], hypertension, coronary artery disease, hyperlipidemia (e.g., LDL, TAGs), hypolipidemia (e.g., HDL), lipid metabolism disorders, lipid deposition disorders, lipodystrophies). In specific embodiments, the invention relates to using a PAT family protein to screen agents for the ability to regulate lipid metabolism and using the same to treat diseases or disorders related to lipid metabolism. In further embodiments, the invention relates to high throughput screening (HTS) methods that can be used in the drug discovery process for screening agents for the ability to regulate lipid metabolism.10-09-2008
20080249048Methods and Compositions for the Treatment of Intestinal Conditions - Methods and compositions for the treatment of intestinal disorders, such as IBD and Crohn's disease, are disclosed. Preferred compositions include siNA. Also disclosed is a method of specifically targeting siNA to treat intestinal disorders by intrarectal administration of siNA compounds.10-09-2008
20080249042All-Trans-Retinol: All-Trans-13,14-Dihydroretinol Saturase and Methods of Its Use - Compositions of all-trans-retinol: all-trans-13,14-dihydroretinal saturase and methods of use thereof are provided.10-09-2008
20080249044Nucleic acid external skin formulation - The present invention provides a nucleic acid external skin formulation having high skin permeability, which is prepared from a polymeric nucleic acid which has high molecular weight and sodium alginate. The nucleic acid external skin formulation is highly skin permeable and delivers its active ingredient nucleic acid to the affected area efficiently. The nucleic acid used may be smaller in amount, and thus, the formulation is also superior in safety and cost. The working mechanism thereof is different from that of the low-molecular weight compounds currently used as an active ingredient for skin anti-inflammatory agents, and thus, the formulation may be applicable to cases where such low-molecular weight medicines are less effective.10-09-2008
20080249056Methods of Altering an Immune Response Induced by Cpg Oligodeoxynucleotides - It is disclosed herein that agents that affect the activity and/or expression of CXCL16 can be used to alter the uptake of D-type CpG oligodeoxynucleotides (D ODNs). Methods of inducing an immune response are disclosed that include administering agents that increase the activity and/or expression of CXCL16 and a D ODN. Methods of decreasing an immune response to a CpG ODN are also disclosed. These methods include administering an agent that decreases the activity and/or expression of CXCL16. Compositions including one or more D-type ODNs and an agent that modulates that activity and/or expression of CXCL16 are provided.10-09-2008
20080249046MODIFIED siRNA MOLECULES AND USES THEREOF - The present invention provides chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression. Advantageously, the modified siRNA of the present invention is less immunostimulatory than its corresponding unmodified siRNA sequence and retains full RNAi activity against the target sequence. The present invention also provides nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided.10-09-2008
20080249039Modified Short Interfering Rna (Modified Sirna) - The present invention is directed to modified siRNA which are significantly impaired in their ability to support cleavage of mRNA when incorporated into a RISC complex. Such modified siRNA may be useful as therapeutic agents, e.g., in the treatment of various cancer forms. More particularly, the modified siRNA comprises a sense strand and an antisense strand, wherein the sense strand contains a modified RNA nucleotide in at least one of positions 8-14, calculated from the 5′-end.10-09-2008
20080249041Formulations comprising antisense nucleotides to connexins - A therapeutic and/or cosmetic formulation comprising at least one anti-sense polynucleotide to a connexin protein together with a pharmaceutically acceptable carrier or vehicle is useful in site specific down regulation of connexin protein expression, particularly in reduction of neuronal cells death, wound healing, reduction of inflammation, decrease of scar formation and skin rejuvenation and thickening.10-09-2008
20080249038Bone Morphogenetic Protein (Bmp) 2A and Uses Thereof - The present invention provides compositions and methods for alleviation or reduction of the symptoms and signs associated with damaged neuronal tissues whether resulting from tissue trauma, or from chronic or acute degenerative changes. In particular, some embodiments of the present invention provide one or more pharmaceutical compositions comprising as an active ingredient a BMP2A inhibitor further comprising a pharmaceutically acceptable diluent or carrier. An additional embodiment provides a method for reducing damage to the central nervous system in a patient who has suffered an injury to the central nervous system, comprising administering to the patient a pharmaceutical composition in a dosage sufficient to reduce the damage. Yet another embodiment provides of the use of a BMP2A inhibitor for the preparation of a medicament for promoting or enhancing recovery in a patient who has suffered an injury to the central nervous system. Preferable inhibitors according to some embodiments of the invention are siRNA molecules and neutralizing antibodies. An additional embodiment provides a method for identifying a chemical compound that modulates apoptosis. Further, a process for diagnosing a neurodegenerative disease or an ischemic event in a subject is provided. The preferred methods, materials, and examples that will now be described are illustrative only and are not intended to be limiting; materials and methods similar or equivalent to those described herein can be used in practice or testing of the invention. Other features and advantages of the invention will be apparent from the following detailed description, and from the claims.10-09-2008
20090131362USE OF DEFIBROTIDE FOR THE INHIBITION OF HEPARANASE - A study has been carried out to verify the effect of Defibrotide on the activity and expression of Heparanase enzyme on myeloma tumor cells (U266) and human microvascular endothelial cells (HMEC). The study has demonstrated that defibrotide can be effectively used for the manufacture of a medicament for the treatment of diseaseses which are positively affected by the inhibition of Heparanse and/or by the downregulation of Heparanse gene expression, such as diabetes.05-21-2009
20090029935Centrosomal proteins and secretion - Described are methods for modulating cellular secretion, and methods for identifying novel modulators of cellular secretion, that target centrosomal proteins.01-29-2009
20090029938OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATING DISEASE INCLUDING INFLAMMATORY CONDITIONS - The invention relates to therapeutic antisense oligonucleotides directed against genes coding for phosphodiesterase (PDEs) and the use of these in combination. These antisense oligonucleotides may be used as analytical tools and/or as therapeutic agents in the treatment of disease associated with reduced cellular cAMP in a patient, such as inflammatory diseases of the respiratory tract including, for example, asthma, chronic obstructive pulmonary disease (COPD), acute respiratory distress syndrome, bronchitis, chronic bronchitis, silicosis, pulmonary fibrosis, lung allograft rejection, allergic rhinitis and chronic sinusitis as well as other conditions in which an increase in cyclic AMP or a decrease in PDE levels is beneficial.01-29-2009
20090029936METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.01-29-2009
20080249045MGAL: A GAL Gene Switch-Based Suite of Methods for Protein Analyses and Protein Expression in Multicellular Organisms and Cells Therefrom - The invention provides methods for detecting and analyzing protein-protein interactions and agonists and antagonists thereof, detecting and analyzing protein sequences, and regulatable gene expression in multicellular organisms or cells therefrom.10-09-2008
20090118212METHODS FOR PRODUCING AND USING IN VIVO PSEUDOTYPED RETROVIRUSES - The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.05-07-2009
20090118219FLEA GABA RECEPTOR SUBUNIT NUCLEIC ACID MOLECULES - The present invention relates to flea GABA receptor subunit nucleic acid molecules; to flea GABA receptor subunit proteins encoded by such nucleic acid molecules; to antibodies raised against such proteins; and to compounds that inhibit the activity of such proteins. The present invention also includes methods to obtain such proteins, nucleic acid molecules, antibodies, and inhibitory compounds. The present invention also includes therapeutic compositions comprising such proteins, nucleic acid molecules, antibodies and inhibitory compounds, particularly those that specifically inhibit flea GABA receptor subunit activity, as well as the use of such therapeutic compositions to treat animals.05-07-2009
20090118210Epididymal lipocalin gene and uses thereof - Isolated nucleic acids comprising a lipocalin gene promoter region, isolated nucleic acids comprising a human lipocalin gene, isolated nucleic acids encoding a lipocalin polypeptide, isolated lipocalin polypeptides, and uses thereof. The disclosed lipocalin nucleic acids and polypeptides can be used to generate a mouse model of male infertility, for drug discovery screens, and for therapeutic treatment of fertility-related conditions.05-07-2009
20090118205Antisense Oligonucleotides Directed to Ribonucleotide Reductase R2 and uses Thereof in the Treatment of Cancer - The present invention provides antisense oligonucleotides directed to a mammalian ribonucleotide reductase R2 gene and combinations of the antisense oligonucleotides with one or more chemotherapeutic agents for use in the treatment of cancer. 05-07-2009
20090118207Use of apoptosis-specific eIF-5A siRNA to down regulate expression of proinflammatory cytokines to treat sepsis - The present invention relates to apoptosis specific eucaryotic initiation factor 5A (eIF-5A), referred to as apoptosis-specific eIF-5A or eIF5-A1, nucleic acids and polypeptides and methods for down regulating pro-inflammatory cytokines in a mammal by administering siRNA against eIF-5A1 to the mammal to treat/prevent sepsis and/or hemorrhagic shock.05-07-2009
20090111765COMPOSITIONS AND METHODS FOR IMMUNOSTIMULATORY RNA OLIGONUCLEOTIDES - The present invention provides 4-nucleotide (4mer) RNA motifs that confer immunostimulatory activity, in particular, IL-12-inducing activity to a single-stranded RNA oligonucleotide. The present invention also provides single-stranded RNA oligonucleotides, including antisense RNA, with high or low immunostimulatory activity. The present invention further provides the use of the RNA oligonucleotides of the invention for therapeutic purposes.04-30-2009
20090105175Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.04-23-2009
20090082288METHODS AND COMPOSITIONS FOR THE TREATMENT OF UVEITIS - Pharmaceutical compositions are disclosed that are of use for the treatment of uveitis. These compositions include a suppressive oligonucleotide. These compositions including an immunosuppressive oligonucleotide can be used for the treatment of uveitis, including anterior, posterior, and diffuse uveitis.03-26-2009
20090082293TECHNIQUES AND COMPOSITIONS FOR TREATING CARDIOVASCULAR DISEASE BY IN VIVO GENE DELIVERY - Methods are provided for treating patients with cardiovascular disease, including heart disease and peripheral vascular disease. The preferred methods of the present invention involve in vivo delivery of genes, encoding angiogenic proteins or peptides, to the myocardium or to peripheral ischemic tissue, by introduction of a vector containing the gene into a blood vessel supplying the heart or into a peripheral ischemic tissue.03-26-2009
20090082290Intra-vascular kidney gene therapy with plasmid encoding BMP-7 - The present invention relates to recombinant vectors expressing the BMP-7 polypeptide in host cells and to pharmaceutical compositions comprising such recombinant vectors. The invention also encompasses methods for prevention and/or treatment of both acute and chronic renal failure in mammals, advantageously in humans, dogs and cats, by intra-vascular kidney administration of the recombinant vectors and pharmaceutical compositions of the invention.03-26-2009
20090054368Substituted gemini surfactant compounds - The compositions, systems, and methods relate to substituted and asymmetric gemini surfactants for use in delivering biologically active agents, such as nucleic acids, to a subject. The described compositions, systems, and methods are useful for gene therapy.02-26-2009
20080242630Fusion proteins of mycobacterium tuberculosis - The present invention relates to compositions and fusion proteins containing at least two 10-02-2008
20080242625Nucleotide-Cochleate Compositions And Methods Of Use - The present invention is directed to cochleate composition that include a nucleotide. The nucleotide may generally be bound via a linker to a component of the cochleate, or to a lipophilic tail. Additionally or alternatively, the nucleotide may be associated with a transfection agent. The present invention also includes methods for making and using the compositions provided herein.10-02-2008
20080242624Cyclin D Polynucleotides, Polypeptides and Uses Thereof - The invention provides isolated polynucleotides, specifically Cyclin D polynucleotides, and their encoded proteins that are involved in cell cycle regulation. The invention further provides recombinant expression cassettes, host cells, transgenic plants, and antibody compositions. The present invention provides methods and compositions relating to altering cell cycle protein content and/or composition of plants for the purpose of increasing transformation efficiency.10-02-2008
20080242626End-Modified Poly(beta-amino esters) and Uses Thereof - Poly(beta-amino esters) are end-modified to form materials useful in the medical as well as non-medical field. An amine-terminated poly(beta-amino ester) is reacted with an electrophile, or an acrylate-terminated poly(beta-amino ester) is reacted with a nucleophile. The inventive end-modified polymers may be used in any field where polymers have been found useful including the drug delivery arts. The end-modified polymers are particularly useful in delivery nucleic acids such as DNA or RNA. The invention also provides compositions including the inventive end-modified polymers, methods of preparing the inventive polymers, and method of using the inventive polymers.10-02-2008
20080242631Impaired wound healing compositions and treatments - Connexin modulation for the treatment of wounds that do not heal at expected rates, including delayed healing wounds, incompletely healing wounds, and chronic wounds, and associated methods, compositions and articles.10-02-2008
20080293656PROCESSING NUCLEIC ACID - The present invention relates to a method of processing nucleic acid. More particularly, it relates to a method of purifying extra-chromosomal DNA by removing cell debris and/or RNA from a process stream comprising extra chromosomal DNA and a precipitate resulting from preceding cell lysis and/or precipitation reactions. It also relates to nucleic acid, particularly extra chromosomal DNA, purified by a method of the invention; a pharmaceutical composition comprising or consisting of the same and apparatus for said method.11-27-2008
20080274995Suppression of Nuclear Factor-KappaB Dependent Processes Using Oligonucleotides - Antisense oligonucleotides which hybridize with nuclear factor-κB (NF-κB) mRNA and methods of using these oligonucleotides.11-06-2008
20080269156Inhibitors of RTP801 and their use in disease treament - The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases respiratory conditions and hearing disorders based upon inhibition of the RTP801 gene and/or protein.10-30-2008
20080214483Antisense-oligonucleotides for the treatment of immuno-suppressive effects of transforming growth factor-beta (TGF-beta) - Antisense-oligonucleotides or effective derivatives thereof hybridizing with an area of a gene coding for transforming growth factor-β (TGF-β) comprising the following nucleic acid sequences identified in the sequence listing under SEQ ID NO. 1-56 and 137 or comprising the following nucleic acid sequences identified in the sequence listing under SEQ ID NO. 57 to 136 each of the nucleic acids having a DNA- or RNA-type structure.09-04-2008
20080255064Methods for modulating cholecystokinin expression - The invention provides a method for upregulating cholecystokinin (CCK) expression in mammalian pancreatic islets by administrating a CCK upregulating agent. The increased CCK expression activates islet cell proliferation triggering an increase in pancreatic β-cell mass and plasma insulin levels. Accordingly, methods to produce a replenishable supply of islet cells and to ameliorate the symptoms associated with diabetes are also disclosed.10-16-2008
20080255065Small Interfering Rna Molecules Against Ribonucleotide Reductase and Uses Thereof - Small interfering RNA (siRNA) molecules that target a mammalian ribonucleotide reductase gene, and which are capable of inhibiting the expression of their target gene are provided. The siRNA molecules of the invention are capable of attenuating neoplastic cell growth and/or proliferation in vitro and in vivo and, therefore, can be used to attenuate the growth and/or metastasis of various types of mammalian cancers.10-16-2008
20080255066ANTISENSE OLIGONUCLEOTIDE STRATEGIES FOR THE ENHANCEMENT OF CANCER THERAPIES - Effective combinations of antisense agents directed against thymidylate synthase mRNA are provided for use in cancer therapies. Combinations of antisense agents have enhanced activity compared to the activity of the individual antisense agents when used alone. The combinations may be used in conjunction with one or more chemotherapeutic agents to enhance the effects of the chemotherapeutic(s). Such antisense agent combinations constitute improved antisense therapies with application to a variety of cancers or proliferative disorders, including drug resistant cancers.10-16-2008
20080207544Compositions And Methods For Delivery Of Agents Into Cells - Disclosed herein are novel compositions for delivery of biologically active agents into cells comprising a cationic amphiphile and a complementary lipid composition. Also disclosed are methods of making and using such novel compositions.08-28-2008
20090012021Delivery of Sirna by Neutral Lipid Compositions - The present invention relates to the fields of molecular biology and drug delivery. In certain embodiments, the present invention provides methods for the delivery of a siNA (e.g., a siRNA) to a cell via a neutral (non-charged) liposome. These methods may be used to treat a disease, such as cancer.01-08-2009
20090176729METHOD OF TREATING NEURODEGENERATIVE DISEASE - Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.07-09-2009
20090176725CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g., miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.07-09-2009
20090176726METHODS FOR TREATING MITF-RELATED DISORDERS - Methods for treating melanoma and other MITF-related disorders by administering a compound that causes an increase in HIF-1 level or activity (e.g., by increasing the level of HIF-1I in a cell) within cells. Such methods include administration of a compound that is a hydroxylase inhibitor, e.g., a prolyl hydroxylase inhibitor that reduces hydroxylation of HIF-1I thereby causing an increase in HIF-1I in the cell. Such treatment can lead to a decrease in MITF activity or expression.07-09-2009
20090093425TRANSDUCIBLE DELIVERY OF NUCLEIC ACIDS BY REVERSIBLE PHOSPHOTRIESTER CHARGE NEUTRALIZATION PROTECTING GROUPS - This disclosure relates to nucleic acid constructs modified to have a reduced net anionic charge. In some aspects the constructs comprise phosphodiester and/or phosphothioate protecting groups. The disclosure also provide methods of making and using such constructs.04-09-2009
20090012029Cyst Nematode Resistant Transgenic Plants - Compositions and methods for providing cyst nematode resistance are provided. One aspect provides transgenic plants or cells comprising an inhibitory nucleic acid specific for one or more cyst nematode esophageal gland cell polypeptides. Other aspects provide transgenic plants or cells resistant to at least two different cyst nematode species.01-08-2009
20090042826Use of the AXL receptor for diagnosis and treatment of renal disease - The invention is directed to a process of identifying a compound capable of inhibiting the activity of a human Axl receptor that comprises contacting the Axl receptor or cells expressing the Axl receptor with the compound; measuring the Axl receptor activity in the presence of the compound; and comparing the activity measured to that measured in the absence of the compound under controlled conditions, wherein a decrease identifies the compound as being capable of inhibiting the activity. Therapeutic and diagnostic applications are also described.02-12-2009
20090181910METHOD OF PREVENTION AND ALLEVIATION OF TOXICITY BY MODULATION OF IRF3 - The invention provides compounds, compositions, animal models, drug screening methods, pharmaceutical compositions, and methods of treatment which relate to the modulation of the metabolism of xenobiotic compounds by administering agents which act on IRF3 or an IRF3 control pathway to modulate the activity, expression, or levels of cytochrome P450 enzymes involved in the metabolism of xenobiotic compounds in a subject.07-16-2009
20090036394GRP 146 Receptor - We disclose GPR146 G-protein coupled receptor (GPCR) polypeptides comprising the amino acid sequence shown in SEQ ID NO. 3 or SEQ ID NO: 5, and homologues, variants and derivatives thereof. Nucleic acids capable of encoding GPR146 polypeptide are also disclosed, in particular, those comprising the nucleic acid sequences shown in SEQ ID No. 1A, SEQ ID No.1 B, SEQ ID No.2 or SEQ ID NO: 4. The GPR146 receptor is useful in the diagnosis and therapy of dyslipidaemias.02-05-2009
20090048200GPR 26 G-Protein Coupled Receptor - We disclose GPR26 G-protein coupled receptor (GPCR) polypeptides comprising the amino acid sequence shown in SEQ ID NO. 3 or SEQ ID NO: 5, and homologues, variants and derivatives thereof. Nucleic acids capable of encoding GPR26 polypeptide are also disclosed. The GPR26 receptor is useful in the diagnosis and therapy of mental illness.02-19-2009
20090156535MicroRNAs for Modulating Herpes Virus Gene Expression - An algorithm for identification of microRNA (miRNA) targets within viral and cellular RNA is disclosed. Also disclosed are essential herpes virus genes whose transcripts contain one or more targets of miRNAs encoded by herpes viruses or by host cells as predicted by the algorithm, and the use of such targets, miRNAs and their derivatives for modulating viral replication and latency.06-18-2009
20090163431COMPOSITIONS AND METHODS FOR MODULATION OF PDX-1 - Methods and compositions for inhibiting PDX-1 are provided according to the present invention. An anti-PDX-1 agent included in inventive methods and compositions includes an antibody, an aptamer, an antisense oligonucleotide, a ribozyme and/or an inhibitory compound. Methods of inhibiting PDX-1 expression in a tumor cell are provided by the present invention which include contacting a tumor cell with an effective amount of an anti-PDX-06-25-2009
20090099117RNA INTERFERENCE MEDIATED INHIBITION OF MYOSTATIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating myostatin (GDF8) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of myostatin gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of myostatin genes.04-16-2009
20090048198COMPOSITIONS AND METHODS FOR NUCLEIC ACID DELIVERY SYSTEMS - The present invention relates to compositions and methods for use in delivering nucleic acids and other agents into cells and tissues. In particular, the present invention provides lipid mixtures at the gel-liquid crystalline phase transition providing superior lipofection activity for transferring materials into cells and tissues.02-19-2009
20090118214Compositions for conferring tolerance to viral disease in social insects, and the use thereof - Compositions and methods for reducing susceptibility to infectious disease in bees using RNA interference technology, and more particularly, prevention and treatment of viral infections in honeybees such as Israel acute paralysis virus (IAPV) by feeding of pathogen-specific dsRNA. Further, multiple-pathogen specific dsRNA is disclosed.05-07-2009
20090082301GENE THERAPY FOR RENAL FAILURE - The present invention relates to recombinant vectors expressing the BMP-7 polypeptide in host cells and to pharmaceutical compositions comprising such recombinant vectors. The invention also encompasses methods for prevention and/or treatment of both acute and chronic renal failure in mammals, advantageously in dogs and cats, by administration of the recombinant vectors and pharmaceutical compositions of the invention.03-26-2009
20090186846Methods to reprogram splice site selection in pre-messenger RNAs - The present invention relates to a method of modulating splice site selection, splicing and alternative, the method comprising the step of hybridizing an oligonucleotide-protein conjugate to a target pre-mRNA molecule in a cell or cell extract, wherein the oligonucleotide-protein conjugate comprises an oligonucleotide moiety which comprises at least two distinct sequence elements: (i) a nucleic acid sequence that is complementary to a specific region upstream of the splice site in the target pre-mRNA molecule; and (ii) an extension containing a protein binding site sequence element for covalently binding a protein; wherein the protein moiety comprises a protein capable of modulating splicing of the splice site upon binding with the protein binding site.07-23-2009
20080249047Modulation of Inflammation Through Modulation of Elavl1/HuR Expression - In the present invention modulation of expression of a member of the Elavl1/Hu family is used to modulate at least translation of specific mRNA. Inducible increase of HuR in murine innate compartments suppresses inflammatory responses in vivo. HuR over-expression induced the translational silencing of specific cytokine mRNAs, despite positive or nominal effects on their corresponding turnover. The present invention uses the fact that HuR acts in a pleiotropic fashion in inflammation through its functional interactions with specific mRNA subsets and negative posttransciptional modules.10-09-2008
20080280847PIT-1 AND VASCULAR CALCIFICATION - The present invention relates to methods and compositions comprising small interfering RNAs (siRNAs) to identify nucleotides encoding proteins involved in the co-transport of inorganic phosphate in vascular tissues. Specifically, it relates to the identification of Pit-1 and Pit-2 as key proteins involved in vascular calcification, the deposition of calcium phosphate, in blood vessels. The nucleic acids encoding specific siRNAs and vectors encoding these siRNAs are useful as tools to specifically inhibit the expression of Pit-1, Pit-2, or related proteins in various primary and stably-transformed cell lines, and as tools to explore the roles and functional relationships of these proteins involved in vascular calcification. In addition, they may be useful directly as therapeutic agents in humans delivered to sites of vascular calcification.11-13-2008
20090029937BIODEGRADABLE CATIONIC POLYMER GENE TRANSFER COMPOSITIONS AND METHODS OF USE - The invention provides biodegradable, cationic compositions based on cationic α-amino acid-containing PEA, PEUR and PEU polymers for use in preparation of non-viral gene transfer compositions. In the invention gene transfer compositions a poly nucleic acid is condensed with the polymer to form a soluble unit wherein the electrical charge of the poly nucleic acid is neutralized by the polymers. The invention gene transfer compositions can be used to transfect target cells by contact with the target cells.01-29-2009
20080242628Inhibiting Gene Expression with dsRNA - The present invention relates to the specific inhibition of gene expression in mammals by bringing the target gene into contact with double stranded RNA (dsRNA).10-02-2008
20080293655Novel tandem siRNAS - The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of two or more target genes.11-27-2008
20080269158E2epf Ubiquitin Carrier Protein-Von Hippel-Lindau Interaction and Uses Thereof - The present invention relates to the E2EPF UCP-VHL interaction and the uses thereof, more precisely a method for increasing or reducing VHL activity or level by regulating UCP activity or level to inhibit cancer cell proliferation or metastasis or to increase angiogenesis. The inhibition of UCP activity is accomplished by any UCP activity inhibitor selected from a group consisting of a small interfering RNA (RNAi), an antisense oligonucleotide, and a polynucleotide complementarily binding to mRNA of UCP, a peptide, a peptide mimetics and an antibody, and a low molecular compound. In the meantime, the increase of angiogenesis is accomplished by the following mechanism; UCP over-expression is induced by a gene carrier and thus endogenous VHL is reduced, leading to the stabilization of HIF-10-30-2008
20080312173Treatment of HSV-related pathologies using plasmids that make ssDNA - A composition for treatment of HSV-related pathologies including an expression vector for altering expression of a target sequence in an HSV-infected cell by production of single-stranded cDNA (ssDNA) in the cell in vivo suspended for topical application to an affected site in a suitable delivery vehicle. The expression vector is comprised of a cassette comprising a sequence of interest, an inverted tandem repeat, and a primer binding site 3′ to the inverted tandem repeat, and a reverse transcriptase/RNAse H coding gene, and is transfected into the infected cells for inhibition of HSV replication. The resulting ssDNA binds to the target sequence to alter expression of the target sequence for such purposes as gene activation or inactivation using duplex or triplex binding of nucleic acids, site-directed mutagenesis, interruption of cellular function by binding to specific cellular proteins, or interfering with RNA splicing functions.12-18-2008
20090099108OLIGONUCLEOTIDE DECOYS AND METHODS OF USE - The present invention describes reagents and methods for using a concatemerized double-stranded oligonucleotide molecules (CODN) for transcription factor decoys. In one embodiment, the concatemers consist of a variable number of end-to-end repeated copies of a short (more than 5, 10, 15, 20, 2, 3035, 40, 45, 50, 75, 100, or more by but generally less than about 3 kb) dsDNA containing a sequence or sequences that act as transcription factor decoys. The present invention also provides for the use of the polymers for CODN/polymer complexes to a specific cell type; thus the agent can be made organ, tissue and/or cell-type specific. In another embodiment, the present invention provides for use of the CODN's in vitro or in vivo, in isolated cells or intact animals in which specific blockade of transcription factors or delivery of DNA or other biological effector is desirable. In one embodiment, this includes use as a research tool, including studies of specific genes and studies to identify specific genes regulated by the transcription factors targeted. In another embodiment, the present invention provides for using polyamides for NF-kB-specific CODN delivery in the treatment of myocardial ischemia/reperfusion and myocardial infarction, heart failure and hypertrophy, cardioprotection, stroke, neuroprotection, sepsis, arthritis, asthma, heritable inflammatory disorders, cancer, heritable immune dysfunctions, inflammatory processes, whether caused by disease or injury or infection, oxidative stress to any organ whether caused by disease, surgery or injury. The decoys may be any transcription factors, including, but not limited to, NF-kB, AP-I, ATF2, ATF3, SP 1 and others.04-16-2009
20090069262Cationic Oligonucleotides, Automated Methods for Preparing Same and Their Uses - The invention relates to oligonucleotide-oligocation molecules A03-12-2009
20090075922Nucleic Acid Ligands Which Bind to Hepatocyte Growth Factor/Scatter Factor (HGF/SF) or its Receptor c-met - The invention provides nucleic acid ligands to hepatocyte growth factor/scatter factor (HGF) and its receptor c-met. The nucleic acid ligands of the instant invention are isolated using the SELEX method. SELEX is an acronym for Systematic Evolution of Ligands by EXponential enrichment. The nucleic acid ligands of the invention are useful as diagnostic and therapeutic agents for diseases in which elevated HGF and c-met activity are causative factors.03-19-2009
20090156536AIMP2-DX2 Gene and SiRNA Targeting AIMP2-DX2 - The present invention relates to a variant of AIMP2 lacking exon 2 gene, named as AIMP2-DX2 gene, which is specifically expressed in cancer cells. The AIMP2-DX2 gene and siRNA targeting AIMP2-DX2 can be successfully used in the development of diagnosis and treatment of cancer.06-18-2009
20090149412Method for Inhibition of Phospholamban Activity for the Treatment of Cardiac Disease and Heart Failure - The present invention provides a method for the treatment of heart failure through the use of small peptide complexes and recombinant proteins which function to enhance contractility in failing hearts and reduce blood pressure in individuals with hypertension by inhibiting the interaction between phospholamban and sacroplasmic reticulum Ca06-11-2009
20090082298Methods for producing microRNAs - The invention relates to recombinant vectors for inducible and/or tissue specific expression of double-stranded RNA molecules that interfere with the expression of a target gene. In certain embodiments, the invention relates to the use of Tet (tetracycline)-responsive RNA Polymerase II (Pol II) promoters (e.g., TetON or TetOFF) to direct inducible knockdown in certain cells of an integrated or an endogenous gene, such as p53. The invention also relates to a method for producing transgenic animals (e.g., mice) expressing inducible (such as tetracycline-regulated), reversible, and/or tissue-specific double-stranded RNA molecules that interfere with the expression of a target gene.03-26-2009
20090099118RNA INTERFERENCE MEDIATED INHIBITION OF STEAROYL-CoA DESATURASE (SCD) GENE EXPRESSION USING SHORT INTERFERING NUCELIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating Stearoyl-CoA desaturase (SCD) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of Stearoyl-CoA desaturase (SCD) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of Stearoyl-CoA desaturase (SCD) genes.04-16-2009
20090192113Interfering RNA Duplex Having Blunt-Ends and 3`-Modifications - The present invention relates to double-stranded RNA compounds with at least one blunt end comprising at least one 3′-end of formula07-30-2009
20090124567Influenza Therapeutic - The present invention provides methods and compositions for inhibiting influenza infection and/or replication based on the phenomenon of RNA interference (RNAi) well as systems for identifying effective siRNAs and shRNAs for inhibiting influenza virus and systems for studying influenza virus infective mechanisms. The invention also provides methods and compositions for inhibiting infection, pathogenicity and/or replication of other infectious agents, particularly those that infect cells that are directly accessible from outside the body, e.g., skin cells or mucosal cells. In addition, the invention provides compositions comprising an RNAi-inducing entity, e.g., an siRNA, shRNA, or RNAi-inducing vector targeted to an influenza virus transcript and any of a variety of delivery agents. The invention further includes methods of use of the compositions for treatment of influenza.05-14-2009
20090042825Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer - Compositions, methods, and applications that increase the efficiency of nucleic acid transfection are provided. In one aspect, a pharmaceutical composition may include at least about 0.5 mg/ml concentration of a nucleic acid condensed with a cationic lipopolymer suspended in an isotonic solution, where the cationic lipopolymer includes a cationic polymer backbone having cholesterol and polyethylene glycol covalently attached thereto, and wherein the molar ratio of cholesterol to cationic polymer backbone is within a range of from about 0.1 to about 10, and the molar ratio of polyethylene glycol to cationic polymer backbone is within a range of from about 0.1 to about 10. The composition further may include a filler excipient.02-12-2009
20080234215DNA composition and uses thereof - A plasmid DNA that encodes one or more antigenic genes operably linked to a promoter and a truncated retroviral 3′ LTR exhibits both enhanced safety and acceptable efficiency of expression of antigenic proteins.09-25-2008
20090131354miR-126 REGULATED GENES AND PATHWAYS AS TARGETS FOR THERAPEUTIC INTERVENTION - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-126, using miR-126 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.05-21-2009
20090137506RNA Interference Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase (PDE4B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3. The invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.05-28-2009
20090143327General composition framework for ligand-controlled regulatory systems - The invention provides an improved design for the construction of extensible nucleic acid-based, ligand-controlled regulatory systems, and the nucleic acid regulatory systems resulting therefrom. The invention contemplates improving the design of the switches (ligand-controlled regulatory systems) through the design of an information transmission domain (ITD). The improved ITD eliminates free-floating ends of the switching and the competing strands, and localizes competitive hybridization events to a contiguous strand of competing and switching strands in a strand-displacement mechanism-based switch, thereby improving the kinetics of strand-displacement. The improved regulatory systems have many uses in various biological systems, including gene expression control or ligand-concentration sensing.06-04-2009
20080200410Method for detection of 5T4 RNA in plasma or serum - This invention relates to methods of detecting or inferring the presence of malignant or premalignant cells in a human that express 5T4. Provided are methods for detecting 5T4 RNA in blood, plasma, serum, other bodily fluids, cells, and tissues. The invention thereby provides an aid for the detection, diagnosis, monitoring, treatment, or evaluation of neoplastic disease.08-21-2008
20090005329NUCLEIC ACID BASED LADDER COPOLYMERS - The present invention relates to a copolymer termed a ladder copolymer because it has two backbones that serve as legs/sides of a ladder structure. These two backbones, one of which is a nucleic acid or nucleic acid-like polymer, are linked together as the legs/sides of a ladder are linked together by the rungs.01-01-2009
20090131345Medicament and method for treating recurrent spontaneous abortion - The present invention discloses a pharmaceutical composition for treating a subject with immunological recurrent spontaneous abortion which comprises a therapeutically effective amount of a substance capable of lowering the in vivo level of antinuclear antibody. Particularly, the substance is chromosome No. 2 or fragment thereof containing fibronectin encoding gene derived from the spouse of said subject, or a mixture of chromosome No. 2 or fragment thereof containing fibronectin encoding gene derived from a plurality of males. The present invention also discloses a method for treating immunological recurrent spontaneous abortion.05-21-2009
20090131361Novel Use of MLN51 Gene and Protein - The present invention relates to novel uses of the MLN 51 gene or protein. The MLN 51 gene and protein is closely related to the development of rheumatoid arthritis, particularly chronic synovitis.05-21-2009
20090131359ANTIFIBROTIC THERAPY - The present invention relates to methods of treating severe or rapidly progressing pulmonary fibrosis in a subject in need of a treatment thereof. The methods comprise increasing the activity of pulmonary and activation-regulated chemokine (CCL18) in the lungs of the subject, whereby increasing CCL18 activity modulates the activity of at least one antifibrotic factor in the lungs of the subject. The present invention also relates to methods of screening test procedures that may be capable of treating severe or rapidly progressing pulmonary fibrosis.05-21-2009
20090131360Tripartite RNAi constructs - The present invention provides compositions and methods for inhibiting expression of a target gene in a cell. The process comprises introduction of double-stranded tripartite RNAi constructs into the cells and reducing the expression of the corresponding messenger RNA in the cells. The constructs, which may be packaged in or delivered as sequestered RNAi constructs, differ from the canonical siRNA in that they comprise a tripartite structure which follows the general formula of having (1) an RNAi core (either native or abbreviated), (2) one or more terminal moieties attached to the RNAi core and optionally (3) a linker between the RNAi core and the terminal moiety. Once packaged into sequestration vehicles, the constructs are activated for gene regulation by the application of certain forms of energy05-21-2009
20090131358LOW DENSITY LIPOPROTEIN RECEPTOR-MEDIATED siRNA DELIVERY - The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo. The conjugates comprise a ligand that can bind to a low density lipoprotein receptor (LDLR) or LDLR family member. Therapeutic uses for the conjugates are also provided.05-21-2009
20090131356miR-15, miR-26, miR-31, miR-145, miR-147, miR-188, miR-215, miR-216, miR-331, mmu-miR-292-3P REGULATED GENES AND PATHWAYS AS TARGETS FOR THERAPEUTIC INTERVENTION - The present invention concerns methods and compositions for identifying genes or genetic pathways modulated by miR-15, miR-26, miR-31, miR-145, miR-147, miR-188, miR-215, miR-216, miR-331, mmu-miR-292-3p, and using nucleic acid comprising all or part of the miR-15, miR-26, miR-31, miR-145, miR-147, miR-188, miR-215, miR-216, miR-331, mmu-miR-292-3p sequences to modulate a gene or gene pathway, using this profile in assessing the condition of a patient and/or treating the patient with an appropriate miRNA.05-21-2009
20090131355MULTICISTRONIC VECTORS AND METHODS FOR THEIR DESIGN - Embodiments of the present invention relate to multicistronic vectors and methods for their design. Methods and compositions of the invention include a vector including at least two cistrons, wherein a first cistron includes a first promoter and a first nucleic acid sequence encoding one or more therapeutic agents, and wherein a second cistron comprises a second promoter and a second nucleic acid sequence encoding one or more RNA molecules that interfere with the expression of a biological response modifier or the therapeutic agent, wherein the expression of the first sequence is under control of the first promoter and expression of the second sequence is under control of the second promoter.05-21-2009
20090131353Diagnosis and Treatment of Chronic Lymphocytic Leukemia (CLL) - The present invention provides diagnostic methods and kits for diagnosis of chronic lymphocytic leukemia (CLL) by determining expression levels of isoforms of cyclic nucleotide phosphodiesterases (PDEs) associated with CLL particularly, PDE7B and/or PDE3B, and a ratio of mRNA expression of PDE7B to PDE3B. The present invention provides that CLL lymphocytes uniformly expressed high levels of PDE7B and low levels of PDE3B relative to those of normal lymphocytes. A method of treatment and a pharmaceutical composition for CLL comprising one or more therapeutic agents capable of modulating expression or activity levels of isoforms of PDEs associated with CLL, and/or reversing the ratio of PDE7B/PDE3B mRNA expression levels are also provided.05-21-2009
20090131350Hybrid Hepatocyte Growth Factor Gene Having High Expression Efficiency of Two Heterotypes of Hepatocyte Growth Factor - The present invention relates to a hybrid Hepatocyte Growth Factor (HGF) gene which is prepared by inserting an inherent or foreign intron between exons 4 and 5 in HGF cDNA, which has a base sequence of SEQ ID NO: 2. The gene has high expression efficiency and simultaneously expresses two heterotypes of HGF and dHGF (deleted variant HGF). Further the gene may be used for treating or preventing ischemic or liver diseases.05-21-2009
20090131349Methods and Compositions for Modulating Body Weight and for Treating Weight Disorders and Related Diseases - Methods of modulating body weight and/or fat content of a subject, methods of treating or preventing weight disorders and methods of treating or preventing weight disorder related diseases are disclosed. The methods include modifying an activity or expression of a protein tyrosine phosphatase epsilon (PTPe) so as to modulate the body weight and/or fat content of the subject. Pharmaceutical compositions and articles of manufacture useful in practice of the methods are further disclosed.05-21-2009
20090131348MICRORNAS DIFFERENTIALLY EXPRESSED IN PANCREATIC DISEASES AND USES THEREOF - The present invention concerns methods and compositions for identifying a miRNA profile for a particular condition, such as pancreatic disease, and using the profile in assessing the condition of a patient.05-21-2009
20090131347IMMUNIZATION-FREE METHODS FOR TREATING ANTIGEN-STIMULATED INFLAMMATION IN A MAMMALIAN HOST AND SHIFTING THE HOST'S ANTIGEN IMMUNE RESPONSIVENESS TO A TH1 PHENOTYPE - The invention relates to methods for preventing or reducing antigen-stimulated, granulocytemediated inflammation in tissue of an antigen-sensitized mammal host by delivering an immunostimulatory oligonucleotide to the host. In addition, methods for using the immunostimulatory oligonucleotides to boost a mammal host's immune responsiveness to a sensitizing antigen (without immunization of the host by the antigen) and shifting the host's immune responsiveness to a Th1 phenotype to achieve various therapeutic ends are provided. Kits for practicing the methods of the invention are also provided.05-21-2009
20090131346ANTIBODY AND METHOD FOR IDENTIFICATION OF DENDRITIC CELLS - Method for identifying myeloid or plasmacytoid dendritic cells provided by a mammal, stimulated or unstimulated, comprising the steps of: a) preparing a cell sample; b) contracting the cell sample myeloid or plasmacytoid dendritic cells to form a complex; c) detecting the complex; characterized in that the phosphatase is the Receptor type Tyrosine Phosphatase Gamma Protein (PTPRG), acting as a specific marker of said dendritic cells, and in that the compound is a polypeptide capable of selectively bind to the PTPRG or to a fragment thereof or to a oligonucleotide complementary to a PTPRG mRNA logonucleotide in such a manner as to allow the selective recognizing of the dendritic cells in the cell sample.05-21-2009
20090131351Methods, compositions, and kits for modulating tumor cell proliferation - The present invention relates to compositions, methods, and kits for modulating tumor proliferation using G-rich oligonucleotides and one or more chemotherapeutic agents.05-21-2009
20090131357Compositions and methods for preventing and treating cancer via modulating UBE1L, ISG15 and/or UBP43 - Compositions and methods of using compositions that induce UBE1L or a ubiquitin-like protein ISG15, or inhibit a deconjugase UBP43 to degrade oncogenic proteins and enhance apoptosis of cancer (neoplastic) or pre-cancerous (pre-neoplastic) cells are provided. Methods for the prevention or treatment of cancer via administration of these compositions are also provided.05-21-2009
20090124571METHOD FOR THE SYNTHESIS OF OLIGONUCLEOTIDE DERIVATIVES - Method for the synthesis of nucleotide derivatives wherein molecules of interest are grafted on the oligonucleotide with the help of a “click chemistry” reaction between an azide function on the molecule of interest and an alkyne function on the oligonucleotide, or between an alkyne function on the molecule of interest and an azide function on the oligonucleotide.05-14-2009
20090111764Mitochondrial selection - Systems, methods, compositions and apparatus relating to genome, chromosome, and mitochondria selection are disclosed.04-30-2009
20090099123Antisense microRNA and uses therefor - Provided herein are methods to suppress specificity protein (Sp) activity in a cell associated with a cell proliferative disease. The methods are effective to inhibit a microRNA in the cell using an antisense microRNA oligonucleotide which results in an increase in expression of a specificity protein (Sp) suppressor gene thereby inducing Sp degradation, apoptosis or growth arrest by releasing inhibitors of G2/M (Myt-1) or inhibition. Also provided are methods of treating a cancer using the antisense microRNA oligonucleotide. In addition the present invention provides antisense microRNA-27a oligonucleotides useful in the methods described herein.04-16-2009
20080207542RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.08-28-2008
20090075923Methods of treatment of renal disease - The invention is directed to a process of identifying a compound capable of inhibiting the activity of a human Ax1 receptor that comprises contacting the Ax1 receptor or cells expressing the Ax1 receptor with the compound; measuring the Ax1 receptor activity in the presence of the compound; and comparing the activity measured to that measured in the absence of the compound under controlled conditions, wherein a decrease identifies the compound as being capable of inhibiting the activity. Therapeutic and diagnostic applications are also described.03-19-2009
20090023675RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.01-22-2009
20090018100MATERIALS AND METHODS FOR TREATING OCULAR-RELATED DISORDERS - The present invention is directed to a method of prophylactically or therapeutically treating an animal for at least one ocular-related disorder, e.g., ocular neovascularization or age-related macular degeneration. The method comprises contacting an ocular cell with an expression vector comprising a nucleic acid sequence encoding an inhibitor of angiogenesis and the same or different nucleic acid sequence encoding a neurotrophic agent. The method also can comprise contacting an ocular cell with different expression vectors, each comprising a nucleic acid sequence encoding an inhibitor of angiogenesis and/or a nucleic acid sequence encoding a neurotrophic agent. In addition, the present invention provides a viral vector comprising a nucleic acid sequence encoding pigment epithelium-derived factor (PEDF) or a therapeutic fragment thereof.01-15-2009
20090018096Methods For Regulating The Growth And/Or Survival Of Tumor Cells And Stem Cells By Modulating The Expression Or Function Of The Transcription Factor ATF5 - The present invention provides methods for regulating the growth and/or survival of tumor cells and stem cells by modulating the expression or function of ATF5. The present invention also provides methods for promoting or suppressing differentiation of stem/progenitor cells, for producing differentiated cells and for isolating/purifying differentiated cells, including neural cells. Also provided are differentiated cells, cell populations and transgenic animals comprising same and uses of same. The present invention further provides methods for treating nervous tissue degeneration and for identifying an agent for use in treating nervous tissue degeneration. Methods for promoting apoptosis in neoplastic cells and for treating or preventing tumors, and identifying agents for use in treating or preventing tumors are also provided by the present invention. The present invention further provides methods for identifying agents that inhibit ATF5, agents identified by these methods. Also provided are methods for diagnosing tumors, for assessing the efficacy of therapy to treat tumors and for assessing the prognosis of a subject who has a neural tumor. Finally, the present invention provides a kits for use in detecting and treating tumors.01-15-2009
20090018095Fusion proteins of mycobacterium tuberculosis - The present invention relates to compositions and fusion proteins containing at least two 01-15-2009
20090018094INHIBITION OF BRAIN ENZYMES INVOLVED IN CEREBRAL AMYLOID ANGIOPATHY AND MACULAR DEGENERATION - A method of treating or inhibiting progress of dementia and/or macular degeneration in a mammal involves administering compositions containing siRNA to heme oxygenase-1 (HO-1) or heme oxygenase-2 (HO-2), a matrix metalloproteinase (MMP) inhibitor, a caspase inhibitor, or a metalloporphyrin in a manner that permits access to brain sites and/or the macula of the patient.01-15-2009
20090018093Nucleic Acid Ligands Specific to Immunoglobuline E and Their Use as Atopic Disease Therapeutics - The invention discloses aptamers capable of binding to Immunoglobulin E (“IgE”) useful as therapeutics in and diagnostics of atopic disease and/or other diseases or disorders in which IgE has been implicated. The invention further relates to materials and methods for the administration of aptamers capable of binding to IgE.01-15-2009
20090018092Reducing Nephropathy with Inhibitors of Soluble Epoxide Hydrolase and Epoxyeicosanoids - The invention provides uses and methods for reducing nephropathy in persons with diabetes mellitus (particularly Type 2 diabetes), in persons with metabolic syndrome, in persons with triglyceride levels over 215 mg/dL, and in persons with a cholesterol level over 200 mg/dL, by administering an inhibitor of soluble epoxide hydrolase (“sEH”). Optionally, a cis-epoxyeicosantrienoic acid (“EET”) can be administered with the sEH inhibitor. The invention further provides for using EETs in conjunction with one or more sEH inhibitors to reduce hypertension, and for compositions of EETs coated with a material insoluble in an acid of pH 3 but soluble in a solution with a pH of 7.4 or higher.01-15-2009
20090203635HXAAA01 Polynucleotides - The present invention relates to novel human secreted proteins and isolated nucleic acids containing the coding regions of the genes encoding such proteins. Also provided are vectors, host cells, antibodies, and recombinant methods for producing human secreted proteins. The invention further relates to diagnostic and therapeutic methods useful for diagnosing and treating diseases, disorders, and/or conditions related to these novel human secreted proteins.08-13-2009
20080312169Cosmetic use of D-ribose - D-ribose is incorporated into a cosmetic composition and applied to the skin to reduce the length and area of wrinkles and to improve the complexion of the skin.12-18-2008
20080318892METHODS AND FORMULATIONS FOR PROTECTING CELLS, AND FOR TREATING DISEASES AND CONDITIONS BY OPTIMIZING THE INTRACELLULAR CONCENTRATION OF NAD - Pharmaceutical and cosmetic formulations and methods for optimizing the intracellular concentrations of NAD are provided. The present methods and compounds relate to the use of PBEF, PRPP and various forms of nicotinamide, individually or in combination, for therapeutic, cyto-protective, cosmetic and anti-aging purposes. PBEF, PRPP and nicotinamide, individually or in combination, as administered according to the invention, increase the metabolic fitness, health and performance of the cell, and thereby increase the cell's level of health during its lifecycle. By way of the present formulations and methods, optimizing the intracellular concentration of NAD+ facilitates a balance among the numerous intracellular interactions of NAD+, and its related pathways, such that the health of the cell and its resistance to stress and trauma are increased. This increased robustness attendant to the invention also facilitates the delay of apoptosis.12-25-2008
20080300209GENE EXPRESSION AND PAIN - The present invention relates to double-stranded oligonucleotides, pharmaceutical compositions thereof, and use of such double-stranded oligonucleotides and pharmaceutical compositions to modulate nociceptive signaling in a cell or prevent and/or treat pain in a patient.12-04-2008
20080300210Method of Controlling Insects and Virus Transmission - The present invention provides methods and compositions for controlling insects and virus transmission, including a genetic construct for inhibiting virus transmission by an arthropod, a transgenic plant, plant cell or plant tissue, a method of preventing transmission of an arthropod-dependent viral plant disease, a method of delivering an active toxic fragment of a 12-04-2008
20080300212TREATMENT AND PREVENTION OF HYPERPROLIFERATIVE CONDITIONS IN HUMANS AND ANTISENSE OLIGONUCLEOTIDE INHIBITION OF HUMAN REPLICATION-INITIATION PROTEINS - Antisense oligonucleotides that inhibit expression of human replication- initiation protein as well as methods of preventing or treating hyperproliferative conditions using said oligonucleotides are disclosed. One aspect provides an antisense oligonucleotide that inhibits the expression of human replication-initiation protein and has a sequence complementary to at least a portion of a target sequence encoding a human replication-initiation gene. By administering a therapeutically effective amount of an antisense oligonucleotide or by contacting the hyperproliferating cells with an effective amount of one or more antisense oligonucleotides, expression of replication-initiation protein is inhibited. Methods of screening and testing active antisense oligonucleotides for their ability to inhibit gene expression are also disclosed.12-04-2008
20080300211MICRORNA INHIBITION FOR THE TREATMENT OF INFLAMMATION AND MYELOPROLIFERATIVE DISORDERS - The present disclosure relates to the finding that microRNA-155 plays a role in inflammation, hematopoiesis and myeloproliferation, and that dysregulation of microRNA-155 expression is associated with particular myeloproliferative disorders. Disclosed herein are methods and compositions for diagnosing and treating disorders, including inflammation and myeloproliferation, modulating the levels of expression of one or more genes selected from the group consisting of Cutl1, Arnt1, Picalm, Jarid2, PU.1, Csf1r, HIF1α, Sla, Cepbβ, and Bach1, and the like.12-04-2008
20080300208Isociatrate dehydrogenase and uses therof - The present invention discloses uses for the IDH gene and/or polypeptide and/or modulators thereof in the diagnosis and treatment of apoptosis-related diseases.12-04-2008
20080300204Alpha-Synuclein Antibodies and Methods Related Thereto - Disclosed are antibodies specific for alpha-synuclein conformers and methods related thereto. For example, disclosed are methods of diagnosing a neurodegenerative monitoring a neurodegenerative disease treatment using the disclosed antibodies. Assays, kits, and solid supports related to alpha-synuclein and antibodies specific for alpha-synuclein are also disclosed.12-04-2008
20080300203Stable Quantitation and Detection of Immune Response Levels with Non-Zero Background Peptides - The invention relates to a kit comprising MHC Class I and Class II HLA-coated beads containing specific antigenic peptides for binding to antigen-specific T cells and the appropriate negative control peptides. Also provided are methods for making the coated beads and methods for use. The application of these beads go to the stimulation of peripheral blood cell populations and in vitro-stimulated culture for the elicitation of functional activities such as cell activation and signaling, cytokine secretion, proliferation and cytotoxicity activity.12-04-2008
20080300201Cell Cycle Arrest and Apoptosis - The HIV-1 accessory gene vpr encodes a conserved 96-amino acid protein that is necessary and sufficient for the HIV-1-induced block of cellular proliferation and induction of apoptosis. Expression of vpr in CD412-04-2008
20080300205Epigenetic mechanisms re-establish access to long-term memory after neuronal loss - The invention relates to methods and products for enhancing and improving recovery of lost memories. In particular the methods are accomplished through the increase of histone acetylation.12-04-2008
20090149405Virus-like particle containing a Dengue virus recombinant replicon - The present invention relates virus-like particle vaccine containing dengue virus recombinant replicon as its core and its preparation method. Such vaccine can efficiently express antigen in the infected tells. Because dengue virus can infect dendritic cells, it can efficiently present antigen and has immunity effects. Further, different dengue virus types can be used to repeatedly produce efficient immune response, which strengthen body's immune response against the pathogen containing such antigen. Such vaccine can prevent and cure cancer and viral diseases.06-11-2009
20080287385Inhibitor oligonucleotides and their use for specific repression of a gene - A double-strand oligonucleotide including two complementary oligonucleotide sequences forming a hybrid, each including at one of their 3′ or 5′ ends, one to five unpaired nucleotides forming single-strand ends extending beyond the hybrid, one of the oligonucleotide sequences being substantially complementary to a target sequence belonging to a DNA or RNA molecule to be specifically repressed, the target sequence belonging to a DNA or RNA molecule of a gene coding an angiogenic factor.11-20-2008
20080280845Compositions and Their Uses Directed to Ptpru - Disclosed herein are compounds, compositions and methods for modulating the expression of PTPRU in a cell, tissue or animal. Also provided are methods of active target segment validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of diabetes, obesity, insulin resistance, insulin deficiency, hypercholesterolemia, hyperglycemia, hyperlipidemia, hypertriglyceridemia, hyperfattyacidemia, liver steatosis, steatohepatitis, non-alcoholic steatohepatitis, metabolic syndrome, cardiovascular disease and coronary heart disease by administration of antisense compounds targeted to PTPRU.11-13-2008
20090069260METHOD FOR PRODUCING A NUCLEIC-ACID-CONTAINING COMPLEX PREPARATION - The present invention relates to a method of preparing a nucleic-acid-containing complex formulation which can be sterilized by filtration and administered intravenously to a human, and can retain stability of polynucleotides included in the nucleic-acid-containing complex formulation. The invention also relates to a method of preparing a nucleic-acid-containing complex formulation, comprising the following steps: mixing a solution comprising two separate single-stranded polynucleotides (for example, poly I and poly C) capable of forming a double strand and a solution comprising a cationic carrier or the ingredients thereof to form the cationic carrier, and performing a dispersion treatment on the mixture.03-12-2009
20090082297Compositions and Methods for Regulating Gene Expression - Methods, compositions and kits for selectively increasing the expression of a target gene are provided.03-26-2009
20090054365RNAi-MEDIATED INHIBITION OF AQUAPORIN 1 FOR TREATMENT OF OCULAR NEOVASCULARIZATION - RNA interference is provided for inhibition of aquaporin 1 (AQP1) to treat conditions associated with neovascularization.02-26-2009
20090093427Articular Cartilage Gene Therapy with Recombinant Vector Encoding BMP-7 - The present invention relates to recombinant vectors expressing the BMP-7 polypeptide in host cells and to pharmaceutical compositions comprising such recombinant vectors. The invention also encompasses methods for prevention and/or treatment of osteoarthritis in mammals, advantageously in humans, dogs, horses and cats, by intra-articular administration of the recombinant vectors and pharmaceutical compositions of the invention.04-09-2009
20090298787Dsrna as Insect Control Agent - The present invention relates to methods for controlling pest infestation using double standard RNA molecules. The invention provides methods for making transgenic plants that express the double stranded RNA molecules, as well as pesticidal agents and commodity products produced by the inventive plants.12-03-2009
20080318891Antisense oligonucleotides against thymidylate synthase - Antisense oligonucleotides targeted to sequences in thymidylate synthase (TS) mRNA. In particular the invention relates to antisense oligonucleotides targeted to sequences in the 3′ end of TS mRNA, which are both cytostatic on their own when administered to human tumour cell lines, and which also enhance the toxicity of anticancer drugs. The invention further relates to a combination product comprising an antisense oligonucleotide in combination with an anticancer agent such as Tomudex or pemetrexed and to the use of such a combination product in the treatment of cancer.12-25-2008
20080249058AGENTS THAT REDUCE NEURONAL OVEREXCITATION - The present invention provides methods of identifying candidate agents for treating excitotoxicity-related disorders. The present invention further provides methods for treating excitotoxicity-related disorders.10-09-2008
20080249057Protein kinase C as a target for the treatment of respiratory syncytial virus - The subject invention concerns a method of inhibiting respiratory syncytial virus (RSV) infection in a patient by decreasing the endogenous protein kinase C (PKC) activity within the patient. Preferably, the preventative and therapeutic methods of the present invention involve administration of a PKC inhibitor. The present inventor has determined that decreasing normal endogenous PKC activity is inhibitory to RSV infection of human cells. The subject invention also pertains to pharmaceutical compositions containing a PKC inhibitor and a pharmaceutically acceptable carrier.10-09-2008
20080249053Compositions And Methods For Detecting And Treating Neurological Conditions - The present invention relates to the NIPA-1 proteins and nucleic acids encoding the NIPA-1 proteins. The present invention further provides assays for the detection of NIPA-1 polymorphisms and mutations associated with disease states, as well as methods of screening for ligands and modulators of NIPA-1 proteins.10-09-2008
20080249051Antimicrobial Compounds - The present invention relates to compounds that modulate the shikimate pathway and/or a pathway branching from the shikimate pathway in members of the Amoebida Order. In particular these compounds may be useful in the treatment or prevention of diseases caused or contributed to by members of the Amoebida Order.10-09-2008
20080249049Polycationically Charged Polymer and the Use of the Same as a Carrier for Nucleic Acid - A composition for the delivery of nucleic acid to target cells or tissues, which comprises polycationically charged polymer as a carrier of nucleic acid is provided. Said polycationically charged polymer is a polymer which may comprise a charged polymer segment having a main chain based on poly(amino acid), polysaccharide, polyester, polyether, polyurethane or vinyl polymer and having, as a side chain, a group of formula —NH—(CH10-09-2008
20080249043Methods and Compositions Involving Protein Kinase C-Delta - Disclosed are methods and compositions relating to the use of PKC-δ activators in the treatment of disease.10-09-2008
20080249040RNA interference mediated inhibition of sterol regulatory element binding protein 1 (SREBP1) gene expression using short interfering nucleic acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Sterol Regulatory Element-Binding Protein 1 (SREBP1) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in Sterol Regulatory Element-Binding Protein 1 (SREBP1) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Sterol Regulatory Element-Binding Protein 1 (SREBP1) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate SREBP1 gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits associated with SREBP1 gene expression or activity in a subject or organism.10-09-2008
20090156531Use of Inhibitors of Scinderin and/or Ephrin-A1 for Treating Tumors - The invention relates to the use of inhibitors of the expression or the activity of scinderin and/or of ephrin-A1 inhibitors for increasing the susceptibility of tumor cells to CTL killing. Such inhibitors may be for instance interfering RNAs targeting the scinderin gene and/or interfering RNAs targeting the ephrin-A1 gene.06-18-2009
20080242634Treatment for neuropathic pain due to spinal cord injury - The present invention is drawn to treatment of neuropathic pain due to spinal cord injury. In this regard, the present invention discloses methods and composition to treat neuropathic pain.10-02-2008
20080242627NOVEL RNA INTERFERENCE METHODS USING DNA-RNA DUPLEX CONSTRUCTS - The present invention provides novel compositions and methods for suppressing the function or activity of a targeted gene through a novel intracellular piRNA-mediated RNAi mechanism, using RNA-DNA duplex constructs. The invention further provides novel methods and compositions for generating or producing RNA-DNA duplex agents, whose quantity is high enough to be used for the invention's gene silencing transfection and possibly in therapeutics applications. This improved RNA-polymerase chain reaction (RNA-PCR) method utilizes thermocycling steps of promoter-linked DNA or RNA template synthesis, in vitro transcription and then reverse transcription to bring up the amount of RNA-DNA duplexes up to two thousand folds within one round of the above procedure for using in D-RNAi-directed gene silencing.10-02-2008
20080234222Charge Reversal of Polyion Complexes - An ionic polymer is utilized in “recharging” (another layer having a different charge) a condensed polynucleotide complex for purposes of nucleic acid delivery to a cell. The resulting recharged complex can be formed with an appropriate amount of positive or negative charge such that the resulting complex has the desired net charge.09-25-2008
20080234220Methods and compositions for reducing viral genome amounts in a small target stem cell - Methods and compositions for reducing viral genome amounts in a target cell are provided. In the subject methods, the activity of a miRNA is inhibited in a manner sufficient to reduce the amount of viral genome in the target cell, e.g., by introducing a miRNA inhibitory agent in the target cell. Also provided are pharmaceutical compositions, kits and systems for use in practicing the subject methods. The subject invention finds use in a variety of applications, including the treatment of subjects suffering from a viral mediated disease condition, e.g., an HCV mediated disease condition.09-25-2008
20080234217Inhibitor Nucleic Acids - The present invention provides methods and compositions for attenuating expression of a target gene in vivo. In general, the method includes administering RNAi constructs (such as small-interfering RNAs (i.e., siRNAs) that are targeted to particular mRNA sequences, or nucleic acid material that can produce siRNAs in a cell), in an amount sufficient to attenuate expression of a target gene by an RNA interference mechanism. In particular, the RNAi constructs may include one or more modifications to improve serum stability, cellular uptake and/or to avoid non-specific effect. In certain embodiments, the RNAi constructs contain an aptamer portion. The aptamer may bind to human serum albumin to improve serum half life. The aptamer may also bind to a cell surface protein that improves uptake of the construct.09-25-2008
20080234214Oligonucleotide-Containing Pharmacological Compositions and Their Use - The present invention relates to methods and compositions containing oligonucleotides suitable for administration to humans and other mammals.09-25-2008
20080234212Materials and Methods for Treatment of Allergic Disease - siRNA molecules are disclosed which target the transcription factor STAT6 and repress the expression of STAT6 mRNA, STAT6 protein and STAT6 function in lung cells. The use of STAT6 specific siRNA molecules in the treatment of allergic diseases of the respiratory tract such as asthma and rhinitis is disclosed.09-25-2008
20080227745Methods and compositions for soluble CPG15 - Disclosed herein are compositions of soluble CPG15 and methods for treating conditions of excessive cell death, such as neurological conditions, using such compositions. Compounds that inhibit the activity of soluble CPG15 are also disclosed herein for the treatment of conditions of undesirable cell survival, such as cancer.09-18-2008
20080227744BENZOFURAN DERIVED HIV PROTEASE INHIBITORS - Resistance-repellent and multidrug resistant retroviral protease inhibitors are provided. Pharmaceutical composition comprising such compounds, and methods of using such compounds to treat HIV infections in mammals, are also provided.09-18-2008
20080227743Compositions and Kits for Treating Influenza - Compositions, kits and methods are provided for the treatment or prophylaxis of influenza.09-18-2008
20080227741ZAP-70 EXPRESSION AS A MARKER FOR CHRONIC LYMPHOCYTIC LEUKEMIA / SMALL LYMPHOCYTIC LYMPHOMA (CLL/SLL) - It has been surprisingly found that ZAP-70 expression, both at the protein and mRNA levels, is indicative of clinical subgroups of CLL/SLL patients. In particular, high ZAP-70 expression is indicative of Ig-unmutated CLL/SLL. Methods are provided for discriminating between clinical subgroups of CLL/SLL, by determining whether subjects overexpress ZAP-70 mRNA or protein.09-18-2008
20080227740Compositions and Methods for Elimination of Unwanted Cells - Disclosed are compositions comprising a recombinant nucleic acid vector including a nucleotide sequence encoding a syncytium-inducing polypeptide expressible on a eukaryotic cell surface, and a host cell containing the recombinant vector and expressing the syncytium inducing polypeptide on its cell surface, the vectors and resultant host cells expressing the syncytium inducing polypeptide being useful for selective elimination of unwanted cells.09-18-2008
20080227739COMPOSITIONS AND METHODS FOR THE THERAPY AND DIAGNOSIS OF BREAST CANCER - Compositions and methods for the therapy and diagnosis of cancer, particularly breast cancer, are disclosed. Illustrative compositions comprise one or more breast tumor polypeptides, immunogenic portions thereof, polynucleotides that encode such polypeptides, antigen presenting cell that expresses such polypeptides, and T cells that are specific for cells expressing such polypeptides. The disclosed compositions are useful, for example, in the diagnosis, prevention and/or treatment of diseases, particularly breast cancer.09-18-2008
20080227738Compositions and methods for cell dedifferentiation and tissue regeneration - The present invention provides methods and compositions to dedifferentiate a cell. The ability of the methods and compositions of the present invention to promote the dedifferentiation of differentiated cells, including terminally differentiated cells, can be used to promote regeneration of tissues and organs in vivo. The ability of the methods and compositions of the present invention to promote the dedifferentiation of differentiated cells, including terminally differentiated cells, can further be used to produce populations of stem or progenitor cells which can be used to promote regeneration of tissues and/or organs damaged by injury or disease. Accordingly, the present invention provides novel methods for the treatment of a wide range of injuries and diseases that affect many diverse cell types.09-18-2008
20080227737Type 2 diabetes mellitus genes - Methods and compositions related to novel genes associated with type 2 diabetes mellitus.09-18-2008
20080227736Targeting Pseudotyped Retroviral Vectors - The present invention relates to retroviral vectors, particularly lentiviral vectors, pseudotyped with Sindbis envelope and targeted to specific cell types via a targeting moiety linked to the envelope.09-18-2008
20080227733Method for Treating and Preventing Ischemia-Reperfusion Injury Using Rna Interfering Agent - The present invention is based, at least in part, on the discovery of methods useful in the modulation, e.g., inhibition, of gene expression or protein activity, e.g., apoptosis-related gene expression, e.g., Fas gene expression or cytokine expression, e.g., proinflammatory cytokine expression. In particular, the present invention is based on novel RNA interfering agents, e.g., siRNA in reduction, e.g., prolonged reduction, of apoptosis-related gene expression or cytokine expression in cells. Inhibition of apoptosis-related gene expression or protein activity or cytokine gene expression or protein activity, e.g. by the siRNAs used in the methods of the invention, inhibits ischemia-reperfusion injury.09-18-2008
20080242633Methods of modulating apoptosis and platelet production using variants of cytochrome c - The invention relates to a method for using the gene or protein containing CYCS(Gly42-Ser) to enhance cellular apoptosis. The method can be used to modulate apoptosis to treat conditions associated with an abnormal rate of apoptosis, in particular to treat conditions associated with increased cell growth, for example hyperplasia, hypertrophy, cancer, neoplasia or the like. The invention also relates to the use of the gene or protein containing CYCS(Gly42-Ser) for stimulating platelet release from megakaryocytes, and also to the treatment of thrombocytopenia using the platelets.10-02-2008
20080221059NOVEL TREATMENT TOOL FOR CANCER: RNA INTERFERENCE OF BCAS2 - The present invention provides three BCAS2 related nucleotide sequences. The present invention also provides a composition comprising a nucleotide sequence of small interfering RNA of BCAS2 gene. The present invention further provides a method for treating p53 containing cancer comprising administrating a subject with an effective amount of the said composition.09-11-2008
20080221058Tumor-Specific Vector for Gene Therapy - The invention relates to a vector for the gene therapeutic treatment of tumors, especially in connection with radiotherapy. Said vector is provided with a therapeutic gene in the DNA sequence thereof. The gene is controlled by the promoter for the catalytic subunit of the telomerase or by the promoter for cyclin A.09-11-2008
20080221057SECRETED PROTEIN CCDC80 REGULATES ADIPOCYTE DIFFERENTIATION - Disclosed herein are methods of modulating adipogenesis. The methods include contacting a cell expressing the Ccdc80 gene with an agent that modulates the expression or activity of the Ccdc80 gene or Ccdc80 protein. Further disclosed herein are methods of treating conditions such as obesity, insulin resistance, and/or type 2 diabetes with Ccdc80 modulators. Also disclosed herein are methods of identifying Ccdc80 modulators.09-11-2008
20080221056Early Detection and Prognosis of Colon Cancers - We have developed a transcriptome-wide approach to identify genes affected by promoter CpG island hypermethylation and transcriptional silencing in colorectal cancer (CRC). By screening cell lines and validating tumor specific hypermethylation in a panel of primary human CRC samples, we estimate that nearly 5% of all known genes may be promoter methylated in an individual tumor. When directly compared to gene mutations, we find a much larger number of genes hypermethylated in individual tumors, and much higher frequency of hypermethylation within individual genes harboring either genetic or epigenetic changes. Thus, to enumerate the full spectrum of alterations in the human cancer genome, and facilitate the most efficacious grouping of tumors to identify cancer biomarkers and tailor therapeutic approaches, both genetic and epigenetic screens should be undertaken. The genes we identified can be used inter alia diagnostically to detect cancer, pre-cancer, and likelihood of developing cancer.09-11-2008
20080221055COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HUNTINGTIN GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.09-11-2008
20080221054Inhibiting Gene Expression with dsRNA - The present invention relates to the specific inhibition of gene expression in mammals by bringing the target gene into contact with double stranded RNA (dsRNA).09-11-2008
20080221053RNA APTAMERS AND METHODS FOR IDENTIFYING THE SAME - RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.09-11-2008
20080221052Modulation of Wrn-Mediated Telomere-Initiated Cell Signaling - The use of modulators of WRN is described. Activators of WRN may be used to induce growth arrest, apoptosis or proliferative senescence, whereas inhibitors of WRN may be used to reduce growth arrest, apoptosis or proliferative senescence. Methods of identifying modulators of WRN are also described.09-11-2008
20080221051Formulations comprising antisense nucleotides to connexins - A therapeutic and/or cosmetic formulation comprising at least one anti-sense polynucleotide to a connexin protein together with a pharmaceutically acceptable carrier or vehicle is useful in site specific down regulation of connexin protein expression, particularly in reduction of neuronal cells death, wound healing, reduction of inflammation, decrease of scar formation and skin rejuvenation and thickening.09-11-2008
20080221050Method for Diagnosing or Predicting Susceptibility to Optic Neuropathy - Disclosed is a set of genetic polymorphisms linked to optic neuropathy including glaucoma and Leber's disease. Those polymorphisms are useful for diagnosing and predicting susceptibility to optic neuropathy.09-11-2008
20090143324RNA INTERFERENCE MEDIATED INHIBITION OF INTERLEUKIN AND INTERLEUKIN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of interleukin and/or interleukin receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of interleukin and/or interleukin receptor genes such as IL-1, IL-2, IL-3, IL-4, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL-1, IL-12, IL-13, IL-14, IL-15, IL-16, IL-17, IL-18, IL-19, IL-20, IL-21, IL-22, IL-23, IL-24, IL-25, IL-26, and IL-27 genes and IL-1R, IL-2R, IL-3R, IL-4R, IL-5R, IL-6R, IL-7R, IL-8R, IL-9R, IL-10R, IL-06-04-2009
20080214488TRIGGERED RNAi - The present application relates to methods and compositions for triggering RNAi. Triggered RNAi is highly versatile because the silencing targets are independent of the detection targets. In some embodiments, a method of silencing a target gene is provided. The method comprises providing an initiator to a cell comprising a detection target and a silencing target gene, wherein the detection target is different from the silencing target gene; providing a first substrate monomer to the cell, wherein the first substrate monomer comprises a silencing target complement region that is substantially complementary to a portion of the silencing target gene, and an initiator complement region that is substantially complementary to a portion of the initiator; and providing a second substrate monomer to the cell, wherein the second substrate monomer comprises a silencing target region that is substantially complementary to the silencing target complement region; wherein binding of the detection target to the initiator initiates formation of an inactivator double-stranded RNA (inactivator dsRNA) which silences the silencing target gene.09-04-2008
20080214486RNAi-MEDIATED INHIBITION OF AQUAPORIN 4 FOR TREATMENT OF IOP-RELATED CONDITIONS - RNA interference is provided for inhibition of aquaporin 4 (AQP4) in intraocular pressure-related conditions, including ocular hypertension and glaucoma such as normal tension glaucoma and open angle glaucoma.09-04-2008
20080214485Method of inducing an immune response - A method is provided for inducing or enhancing an immune response in a mammal to a target polypeptide expressed in a plurality of cells of the mammal, which method comprises administering to the mammal an inhibitory nucleic acid which targets a region of a ribonucleic acid (RNA) which encodes said polypeptide. Also provided is a pharmaceutical composition comprising an inhibitory nucleic acid which targets a region of an RNA which encodes a target polypeptide expressed in a plurality of cells of a mammal, such that translation of an aberrant form of the target polypeptide occurs in said cells, said truncated form of the target polypeptide comprising one or more T cell epitopes; together with a pharmaceutically acceptable carrier or diluent.09-04-2008
20090075927SWEET TASTE RECEPTORS - This invention provides novel genes and polypeptides of the sweet receptor family, methods for production of the polypeptides, methods for screening compounds that specifically bind to and/or modulate the activity of these polypeptides; and antibodies specific for the polypeptides.03-19-2009
20080207555HYDROLASE AND METHODS FOR ITS USE - This disclosure provides methods for catalyzing the release of ADP-ribose from poly(ADP-ribose) or O-acetyl-ADP-ribose. Also provided are methods for modifying DNA repair or chromatin structure by introducing into the cell an agent that modifies the activity of an ARH3 polypeptide, or variant or fragment thereof. Further provided are methods for screening molecules involved in the poly(ADP-ribosyl)ation of proteins or O-acetyl-ADP-ribose content, and method for treating disorders by altering activity of an ARH3 protein.08-28-2008
20080200420In vivo production of small interfering RNAs that mediate gene silencing - The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.08-21-2008
20080200419Splicing Variant of TGF-beta2 and Uses Thereof - An alternatively spliced form of transforming growth factor-beta2 (TGF-β2), herein denoted Δ6-TGF-β2 is disclosed. Δ6-TGF-β2 differs from TGF-β2 in the sequence of the three C-terminal exons. This novel protein is secreted, induced by cytotoxic stress in hematopoietic stem cells, and specifically blocks the enhancing effects of TGF-β2 on adult stem cells. Δ6-TGF-β2 can be used to protect stem cells from cytotoxic stress, and to enhance maintenance of these cells in vitro during retroviral transduction. In addition, Δ6-TGF-β2 can be used to slow aging and extend longevity.08-21-2008
20080200418COMPOSITIONS FOR DRUG ADMINISTRATION - A composition having at least one alkyl glycoside and at least one therapeutic agent, wherein the alkylglycoside has an alkyl chain length from about 10 to about 16 carbon atoms and wherein the therapeutic agent is an oligonucleotide.08-21-2008
20080200417HIGH EFFICIENCY ENCAPSULATION OF CHARGED THERAPEUTIC AGENTS IN LIPID VESICLES - Methods for the preparation of a lipid-nucleic acid composition are provided. According to the methods, a mixture of lipids containing a protonatable or deprotonatable lipid, for example an amino lipid and a lipid such as a PEG- or Polyamide oligomer-modified lipid is combined with a buffered aqueous solution of a charged therapeutic agent, for example polyanionic nucleic acids, to produce particles in which the therapeutic agent is encapsulated in a lipid vesicle. Surface charges on the lipid particles are at least partially neutralized to provide surface-neutralized lipid-encapsulated compositions of the therapeutic agents. The method permits the preparation of compositions with high ratios of therapeutic agent to lipid and with encapsulation efficiencies in excess of 50%.08-21-2008
20080200416Modulation of T cell signaling threshold and T cell sensitivity to antigens - MicroRNAs (miRNAs) are a diverse and abundant class of ˜22-nucleotide (nt) endogenous regulatory RNAs that play a variety of roles in animal cells by controlling gene expression at the posttranscriptional level. Increased miR-181a expression in mature T cells is shown to cause a marked increase in T cell activation and augments T cell sensitivity to peptide antigens. Moreover, T cell blasts with higher miR-181a expression become reactive to antagonists. The effects of miR-181a on antigen discrimination are in part achieved by dampening the expression of multiple negative regulators in the T cell receptor (TCR) signaling pathway, including PTPN22 and the dual specificity phosphatases DUSP5 and DUSP6. This results in a reduction in the TCR signaling threshold, thus quantitatively and qualitatively enhancing T cell sensitivity to antigens.08-21-2008
20080200415Plant Activation of Insect Toxin - Compositions and methods for protecting a plant from an insect pest are provided. In particular, nucleic acid sequences encoding insect protoxins modified to comprise at least one proteolytic activation site that is sensitive to a plant protease or an insect gut protease are provided. Cleavage of the modified protoxin at the proteolytic activation site by a protease produces an active insect toxin. Methods of using the modified insect protoxin nucleic acid sequences and the polypeptides they encode to protect a plant from an insect pest are provided. Particular embodiments of the invention further provide modified insect protoxin compositions and formulations, expression cassettes, and transformed plants, plant cells, and seeds.08-21-2008
20080200413RNA APTAMERS AND METHODS FOR IDENTIFYING THE SAME - RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.08-21-2008
20080200412Astrocyte Elevated Gene-1 And Its Promoter In Treatments For Neurotoxicity And Malignancy - The present invention is based, at least in part, on the discovery that Astrocyte Elevated Gene-1 (‘AEG-1’) expression (i) suppresses the Excitatory Amino Acid Transporter-2 (‘EAAT-2’) promoter, thereby inhibiting glutamate transport; (ii) supports anchorage independent colony formation of cells, in which it is synergistic with the RAS oncogene; and (iii) is increased in a number of different malignancies. The invention, in various embodiments, provides for methods of treatment of malignancies and neurodegenerative disorders using inhibitors of AEG-1 activity, and provides for screening assays for identifying other compounds that have therapeutic benefit.08-21-2008
20080200411MEANS AND METHODS FOR DIAGNOSING AND TREATING AFFECTIVE DISORDERS - Nucleic acid molecules encoding an ATP-gated ion channel P2X7R which contains a mutation or a deletion are disclosed. Polypeptides encoded by the nucleic acid molecules and antibodies that specifically are directed to these polypeptides are disclosed. Aptamers that specifically bind the nucleic acid molecules, and primers for selectively amplifying the nucleic acid molecules are provided, kits, compositions, particularly pharmaceutical and diagnostic compositions comprising the nucleic acid molecules, vectors, polypeptides, aptamers, antibodies and/or primers, are provided. Methods for diagnosing affective disorders associated with a non-functional P2X7R protein, an altered ATP-gating of the P2X7R protein, an over or underexpression of the P2X7R protein or associated with the presence of any one of the nucleic acid molecules or polypeptides encoded thereby are disclosed. Additionally, the present invention relates to uses and methods for treating affective disorders employing a functional or non-functional ATP-gated ion-channel P2X7R, such as treatment with modulators of P2X7R activity.08-21-2008
20080200409Antisense Oligonucleotides For Inducing Exon Skipping and Methods of Use Thereof - Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.08-21-2008
20080200408Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit - The present invention is directed to deletion mutants of a human sodium channel alpha subunit and methods and compositions for making and using the same.08-21-2008
20080200407Methylated cpg polynucleotide - It is an object of the present invention to provide a polynucleotide, which can effectively suppress the immunoreactivity caused by DNA having a CpG motif and which can be used for preventing and/or treating immune-mediated diseases such as arthritis. The present invention provides a polynucleotide comprising a CpG motif wherein guanine is methylated, and a pharmaceutical composition comprising the above-mentioned polynucleotide.08-21-2008
20080300202SUBTRACTIVE TRANSGENICS - Described herein are methods for predictable, highly selective expression of a transgene in a human or non-human animal. The methods comprise subtractive transgenics, wherein the expression pattern of one selective promoter is subtracted from the expression pattern of a second selective promoter. Provided are non-human transgenic animals exhibiting a highly selective expression pattern and methods of producing such animals. Further provided are methods of selective expression of a transgene in an animal, including a human, by administration of viral vectors.12-04-2008
20090018097MODIFICATION OF DOUBLE-STRANDED RIBONUCLEIC ACID MOLECULES - A double-stranded RNA (dsRNA) molecule comprising between about 15 base pairs and about 40 base pairs, wherein at least one ribonucleotide of the dsRNA is a 5′-methyl-pyrimidine and/or at least one 2′-O-methyl ribonucleotide, and a method of improving ribonuclease stability, reducing off-target effects of a double stranded siRNA molecule, or of reducing interferon responsiveness of a double stranded siRNA molecule using such dsRNA.01-15-2009
20090093434USE OF EIF-5A1 SIRNA TO PROTECT ISLETS CELLS FROM APOPTOSIS AND TO PRESERVE THEIR FUNCTIONALITY - The present invention relates to methods for improving the viability, recovery and functionality of islets that are separated from a donor organ for subsequent transplantation and more particularly relates to the use of eIF-5A1 siRNAs to enhance the viability and functionality of islets.04-09-2009
20090118211Compositions and Methods for Enhancement of Sexual Function - Novel compositions and methods which utilize an erection-enhancing agent and an ejaculation-delaying agent, such that these agents provide an overlapping and/or synergistic effect, are provided. These composition and methods can be beneficially utilized for treating sexual dysfunction and/or for enhancing sexual function.05-07-2009
20090075921Bone/joint disease sensitivity gene and use thereof - The present invention provides the prophylaxis and treatment of bone and joint diseases by regulating the expression or activity of calmodulin, the prophylaxis and treatment of bone and joint diseases by regulating the expression or activity of asporin, and a diagnostic method for genetic susceptibility to bone and joint diseases by detecting polymorphisms in the CALM1 gene and/or the asporin gene, and the like.03-19-2009
20090163437STEADY-STATE SUBCUTANEOUS ADMINISTRATION OF APTAMERS - An improved method of administration of an aptamer and modulator system to regulate blood coagulation in a host is provided wherein the aptamer is administered subcutaneously and the modulator is administered either subcutaneously or intravenously. This method for sustained aptamer activity using intermittent subcutaneous injections further allows for titrated modulation of the aptamer activity by administration of the modulator.06-25-2009
20090209628MULTIPLE RNAi EXPRESSION CASSETTES FOR SIMULTANEOUS DELIVERY OF RNAi AGENTS RELATED TO HETEROZYGOTIC EXPRESSION PATTERNS - The present invention provides compositions and methods suitable for expressing y-x multiple-RNAi agents against an allele or alleles of interest in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases.08-20-2009
20090209625MODULATION OF CHREBP EXPRESSION - Disclosed herein are compounds, compositions, and methods for modulating the expression of ChREBP in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and conditions.08-20-2009
20090253769Inhibitors of mammalian hdac 11 useful for treating hdac 11 mediated disorders - The present invention, relies, in part, on the discovery of the role of HDAC 11 in various cell proliferative disorders including cancer. In the main, the invention provides for methods of inhibiting HDAC 11 as a means of treating cell proliferative disorders. Reagents for use in inhibiting human HDAC 11 are also provided.10-08-2009
20120172415Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy - Methods for stabilizing unstable proteins or for restoring functionality to non-functional or poorly functioning (semi-functional) proteins using exon skipping technology are provided. The methods involve the administration of antisense oligonucleotides to cause exon skipping, thereby removing one or more exons responsible for protein instability or lack of functionality. For example, exons encoding protease recognition sites may be removed. The method is useful for treating diseases caused by protein instability, such as Becker Muscular Dystrophy, or for treating Duchenne Muscular Distrophy patients with semi-functional dystrophin due to treatment with other exon skipping or stop codon readthrough therapies.07-05-2012
20120172413INCREASING LIFESPAN BY MODULATING CRTC EXPRESSION OR LOCALIZATION, AND METHODS OF SCREENING FOR MODULATORS OF SAME - The invention relates to the field of longevity enhancement. More particularly, the invention provides compositions and methods relating to CRTC modulation. In certain embodiments, the invention provides compositions and methods for enhancing longevity in an organism by inhibiting CRTC activity, such as, for example, inhibiting CRTC expression or cellular localization in the organism.07-05-2012
20120172411NOVEL TRIALKYL CATIONIC LIPIDS AND METHODS OF USE THEREOF - The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.07-05-2012
20120172410SHORT HAIRPIN RNA FOR GENE KNOCKDOWN OF NR1 SUBUNIT OF THE N-METHYL-D-ASPARTATE RECEPTOR AND ITS APPLICATION ON PHARMACEUTICS - A short hairpin RNA (shRNA) for gene knockdown the genetic expression of NR1 subunit of N-methyl-D-aspartate (NMDA) receptor comprises a first fragment sharing homologous nucleotides among the NR1 subunit of NMDA receptor; a second fragment having complementary sequence to the first fragment; and a connecting fragment having any base in repeated arrangement, and connecting to the first and second fragments. Also, a method of treatment for pathological pain, by applying the shRNA described above to subcutaneous tissues of living organisms for gene knockdown the genetic expression of the NR1 subunit of NMDA receptor in hypoderm.07-05-2012
20130046006INHIBITORS OF FAM3B GENE, INHIBITOR COMPOSITIONS, INHIBITING METHODS AND APPLICATIONS OF INHIBITORS IN PREPARING PHARMACEUTICALS - Inhibitors that can inhibit expression of FAM3B gene to reduce the levels of expression products, or can combine the expression products to reduce the activity of promoting lipid synthesis of FAM3B gene product are provided, wherein the inhibitors are one or more inhibitors selected from the group consisting of small interfering RNAs, antisense oligonucleotides, antibodies against FAM3B proteins and active organic compounds. Cells, vectors or inhibitor compositions, comprising such inhibitors, methods for inhibiting expression of FAM3B gene or inhibiting the activity of promoting lipid synthesis of FAM3B gene product using the inhibitors are provided. Methods for treating diseases mediated by expression of FAM3B gene using such inhibitors and uses of the inhibitors in preparing pharmaceuticals for preventing and/or treating the disease mediated by FAM3B gene expression are also provided.02-21-2013
20100016408MODULATING THE CDC14B-CDH1-PLK1 AXIS AND METHODS FOR SENSITIZING TARGET CELLS TO APOPTOSIS - The invention relates to modulating Cdc14B levels (cell division cycle 14 homolog B) and/or Cdh1 (Fzr1 protein, CDC20-like 1b, or fizzy-related protein) levels to sensitize cells to DNA damage by increasing the abundance of Plk1 (polo-like kinase 1) in a target cell. In certain embodiments, the invention relates to modulating Plk1 levels, and in particular to increasing Plk1 levels, to sensitize target cells such as cancer cells to cell death or apoptosis. In certain embodiments, the invention relates to inhibitors of Cdc14B and Cdh1 that sensitize tumor cells to chemotherapy or radiation induced cell death or apoptosis. In addition to applications relating to cancer therapies and diagnostics, the Plk1 modulators and assays will be employed for identifying novel drugs or drug candidates useful for various proliferative and/or differentiative disorders such as major opportunistic infections, immune disorders, cardiovascular diseases and inflammatory disorders.01-21-2010
20120184599Use of MicroRNA for Treating Diseases Associated with a Dysfunction of the Cilia in Multiciliated Epithelial Cells - The present invention relates to a method for evaluating the regenerative and/or differentiation capacity of ciliated epithelial tissue in a vertebrate subject, in particular a mammal, preferably a human, and to the use of microRNA in treating illnesses associated with a dysfunction of multiciliated epithelial cells.07-19-2012
20120184598POLYNUCLEOTIDES FOR MULTIVALENT RNA INTERFERENCE, COMPOSITIONS AND METHODS OF USE THEREOF - The present invention includes bivalent or multivalent nucleic acid molecules or complexes of nucleic acid molecules having two or more target-specific regions, in which the target-specific regions are complementary to a single target gene at more than one distinct nucleotide site, and/or in which the target regions are complementary to more than one target gene or target sequence. Also included are compositions comprising such nucleic acid molecules and methods of using the same for multivalent RNA interference and the treatment of a variety of diseases and infections.07-19-2012
20120184597OLIGORIBONUCLEOTIDES AND METHODS OF USE THEREOF FOR TREATMENT OF ALOPECIA, ACUTE RENAL FAILURE AND OTHER DISEASES - The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a human p53 gene. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating a patient suffering from alopecia or acute renal failure or other diseases comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The alopecia may be induced by chemotherapy or radiotherapy, and the patient may be suffering from cancer, in particular breast cancer.07-19-2012
20120184596MICRORNA INHIBITORS COMPRISING LOCKED NUCLEOTIDES - The invention provides chemically modified oligonucleotides capable of inhibiting the expression (e.g., abundance) of miR-208 family miRNAs, including miR-208a, miR-208b, and/or miR-499. The invention provides in some embodiments, oligonucleotides capable of inhibiting, in a specific fashion, the expression or abundance of each of miR-208a, miR-208b, and miR-499. The invention further provides pharmaceutical compositions comprising the oligonucleotides, and methods of treating patients having conditions or disorders relating to or involving a miR-208 family miRNA, such as a cardiovascular condition. In various embodiments, the oligonucleotides provide advantages in one or more of potency, efficiency of delivery, target specificity, toxicity, and/or stability.07-19-2012
20120184595COMPOSITIONS AND METHODS FOR SILENCING APOLIPOPROTEIN C-III EXPRESSION - The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA that target apolipoprotein C-III (APOC3) gene expression, lipid particles comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles (e.g., for the treatment of lipid diseases or disorders such as atherosclerosis or a dyslipidemia such as hypertriglyceridemia or hypercholesterolemia).07-19-2012
20120184600Inhibition of Glycerol-3-Phosphate Acyltransferase (GPAT) and Associated Enzymes for Treatment of Viral Infections - A method of treating or preventing a viral infection in a mammal by administering a compound or pharmaceutically acceptable derivative thereof that inhibits a phosphatidic acid synthesis enzyme07-19-2012
20110190377METHOD FOR PRODUCING A CELL AND/OR TISSUE AND/OR DISEASE PHASE SPECIFIC MEDICAMENT - A DNAzyme is disclosed, which comprises: 08-04-2011
20090215860COMPOSITIONS AND METHODS FOR REGULATING GENE TRANSCRIPTION - The invention is directed to compositions and methods for RNA-mediated gene regulation, e.g., transcription regulation, e.g., by transcriptional silencing of genes. In one aspect, the invention provides methods using siRNAs directed at a transcription regulator, e.g., a promoter or enhance sequence, of a gene target molecule. In one aspect, this results in in vivo DNA methylation and/or modification of associated chromatin (e.g., histone proteins) accompanied by and/or partial or complete transcription gene silencing in a cell, such as a mammalian cell, e.g., a human cell.08-27-2009
20100152278RNA Interference Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase (PDE4B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-1, IL-8, IL-15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-1, MMP -2, MMP-3, MMP-9 and MMP-12. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), such as endogenous micro-RNA (miRNA) (e.g., miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC) including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.06-17-2010
20090326042COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF CRP - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.12-31-2009
20110196019Treatment of Cancer by Inhibition of IGFBPs and Clusterin - Agents that reduce the amount of IGFBP-2 and/or IGFBP-5 and that are known to be useful in the treatment of cancer result in increased expression of the protein clusterin. Since clusterin can provide protection against apoptosis, this secondary effect detracts from the efficacy of the therapeutic agent. In overcoming this, the present invention provides a combination of therapeutic agents that is useful in the treatment of cancer. The combination includes an agent that reduces the amount of IGFBP-2 and/or IGFBP-5 and that stimulates expression of clusterin as a secondary effect, and an oligonucleotide that is effective to reduce the amount of clusterin in cancer cells. In some embodiments of the invention, the agent that reduces IGFBP-2 and/or IGFBP-5 is a bispecific antisense species. The oligonucleotide may be an antisense oligonucleotide or an RNAi oligonucleotide.08-11-2011
20110196018Nuclease resistant external guide sequences for treating inflammatory and viral related respiratory diseases - External Guide Sequence (EGS) are described that target proteins required for generation and modification of the immunoglobulin and T-cell repertoire that are useful for treatment or prevention of inflammatory or related diseases. Formulations suitable for administration of an EGS for treatment of inflammatory or related disease are described. The formulations may be administered via inhalation, injection, or orally. The formulations may be in the form of an ointment, lotion, cream, gel, drop, suppository, spray, liquid, powder, granule, solution, suspension, capsule, or tablet. Methods of treating inflammatory or related diseases by administering an effective amount of an EGS in a pharmaceutically acceptable carrier are also described. In preferred embodiments, the disease is asthma, allergic rhinitis, food allergies, atopic skin disease such as eczema, IL-4 and/or IL-13 dependent malignancies, IL-4 and/or IL-13 dependent autoimmune diseases, atopic diseases, the flu, and diseases caused by IL-4 dependent replication of viruses.08-11-2011
20110196017MICRO-RNA THAT PROMOTES VASCULAR INTEGRITY AND USES THEREOF - The present invention relates to the identification of a microRNA, designated miR-126, that is a regulator of vascular integrity in endothelial cells. This endothelial cell-restricted microRNA mediates developmental angiogenesis in vivo, and targeted deletion of miR-126 in mice causes leaky vessels, hemorrhaging, and partial embryonic lethality, due to a loss of vascular integrity and defects in endothelial cell proliferation, migration, and angiogenesis. These vascular abnormalities resemble the consequences of diminished signaling by angiogenic growth factors, such as VEGF and FGF. These findings have important therapeutic implications for a variety of disorders involving abnormal angiogenesis and vascular leakage. Methods of treating disease states characterized by ischemia, vascular damage, and pathologic neovascularization by modulating miR-126 function are disclosed.08-11-2011
20110196016Compositions and Methods for Inhibiting Expression of IKK2 Genes - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an IKK2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of an IKK2 gene using said pharmaceutical composition; and methods for inhibiting the expression of IKK2 in a cell.08-11-2011
20090221670Method for diagnosis and treatment of a mental disease - The present invention relates to association of one or more polymorphisms located in the human NHP2L1, PACSIN2, SERHL, PIPPIN, BRD1, EP300, FAM19A5 and/or GPR24 genes to the occurrence of schizophrenia and/or bipolar disorder. The invention relates both to methods for diagnosing a predisposition to said diseases and for treating subjects having said diseases.09-03-2009
20130085173SUPERCOILED MINICIRCLE DNA FOR GENE THERAPY APPLICATIONS - The present invention relates to nucleic acid molecule compositions comprising minivectors encoding a nucleic acid sequence and methods of gene therapy and prophylaxis against infection using minivectors encoding a nucleic acid sequence.04-04-2013
20090286851Compositions and Methods for Delivering RNAI Using Lipoproteins - This invention relates to new compositions comprising at least one of a single or double stranded oligonucleotide, where said oligonucleotide has been conjugated to a lipophile and to which the conjugated oligonucleotide has been preassembled with lipoproteins. These compositions are effectively in delivering oligonucleotides to mammalian tissue where they effect gene silencing.11-19-2009
20100048674Role of miRNA in T cell leukemia - The ability of miR-181a to support active signaling between Notch and pre-TCR pathways by coordinately dampening negative regulators of these pathways allows the use of miR-181a as a therapeutic target for T-ALL.02-25-2010
20100113564RNA Interference Mediated Inhibition of Interleukin and Interleukin Receptor Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of interleukin and/or interleukin receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of interleukin and/or interleukin receptor genes.05-06-2010
20100113562THERAPEUTIC TARGETS AND MOLECULES - The invention provides methods and compositions for treating or preventing inflammation or an inflammatory condition in a subject, comprising administering to the subject an effective amount of at least one antagonist of one or more miRNA upregulated in inflammatory disease conditions and response to allergen challenge. The invention also provides methods for diagnosing inflammatory conditions based on miRNA expression profile signatures.05-06-2010
20100113561MICRORNA MOLECULES05-06-2010
20100113559Chitosan Based Polymer Conjugate and a Method for Producing the Same - The present invention relates to a conjugate of chitosan and polyamine polymer that is useful for transferring a desired gene medicine into cells, and a method for preparing the same. In particular, the present invention relates to a double conjugate that is prepared by 1 in king poly-L-arginine to low molecular weight chitosan or triple conjugate that is prepared by additionally linking polyethylene glycol (PEG) to the double conjugate, and a method for preparing the same. The chitosan based cationic polymer conjugate of the present invention forms a complex with negatively charged gene medicine such as plasmid DNA and small interfering RNA to efficiently transfer the desired gene medicine into cells with low cytotoxicity. Accordingly, the conjugate can be used as an effective delivery system for in vivo administration of gene medicine.05-06-2010
20090281166Treatment of Cancer by Inhibition of HSP27 - A cancer is evaluated for selection of appropriate therapy by evaluating a sample of cancerous tissue to determine an expression of level of phosphatase and tensin homologue deleted from chromosome 10 (PTEN); and in the case where the expression level of functional PTEN is below a threshold level, identifying the cancer as susceptible to an active agent that inhibits the expression of heat shock protein 27 (hsp27). The evaluation may be included as part of a method of treatment, in which an hsp27 inhibitor is selected as a therapeutic agent when the PTEN level is below the threshold.11-12-2009
20100076058Methods for identifying analgesic agents - The present invention relates to the discovery that mutations in SCN9A are causative of Congenital Indifference to Pain (CIP) in humans. The invention also relates to methods of utilizing the SCN9A gene and expression products thereof for the screening and identification of therapeutic agents, including small organic compounds, which are selective for SCN9A, and are useful in the treatment of pain and other disorders. The invention also relates to methods of using these compounds to treat or otherwise ameliorate such disorders.03-25-2010
20100076057TARGET DNA INTERFERENCE WITH crRNA - The present invention provides methods, systems, and compositions for interfering with the function and/or presence of a target DNA sequence in a eukaryotic cell (e.g., located in vitro or in a subject) using crRNA and CRISPR-associated (cas) proteins or cas encoding nucleic acids. The present invention also relates to a method for interfering with horizontal gene transfer based on the use of clustered, regularly interspaced short palindromic repeat (CRISPR) sequences.03-25-2010
20100076056MODIFIED iRNA AGENTS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.03-25-2010
20100076055Cationic Lipids and Uses Thereof - Cationic lipids, cationic lipid based drug delivery systems, ways to make them and methods of treating diseases using them are disclosed.03-25-2010
20100076054SENSIZITATION OF CANCER CELLS TO THERAPY USING SINA TARGETING GENES FROM THE 1P AND 19Q CHROMOSOMAL REGIONS - The invention relates to the identification of genes involved in resistance of cancer cells to therapy, to short nucleic acid molecules which inhibit the expression of these genes by RNA interference and to their use as adjuvant in cancer therapy, to sensitize cancer cells to conventional anticancer agents; the short nucleic acid molecules are double-stranded short interfering nucleic acid molecules including a sense and an antisense region, wherein the sense region includes a nucleotide sequence that is selected from the group consisting of: the sequences SEQ ID NO: 15, 11, 13, 14, 30, 31, 38, 46, 64 and 70 and the sequences having at least 70% identity, preferably at least 80% identity, more preferably at least 90% identity with the sequences, and the antisense region includes a nucleotide sequence that is complementary to the sense region.03-25-2010
20100076053SEQUENCE OF DSRNA: ATN-RNA, INTERVENTION USING IRNAI, USE OF A SEQUENCE OF DSRNA: ATN-RNA, A METHOD OF TREATING AND INHIBITING A BRAIN TUMOR, A KIT FOR INHIBITING CANCER CELL WHICH EXPRESSES TENASCIN, A METHOD FOR A KIT PREPARATION IN A BRAIN TUMOR THERAPY - The subject matters of this invention are a sequence of double-stranded RNA: ATN-RNA, intervention using interference RNA (iRNAi), use of a sequence of double-stranded RNA: ATN-RNA, a method of treating a brain tumor and a method of inhibiting a brain tumor cells which express tenascin, a kit for inhibiting cancer cell which expresses tenascin and a method for a kit preparation in a brain tumor therapy. Malignant gliomas preferentially express a number of surface markers that may be exploited as therapeutic targets, including tenascin-C, an extracellular matrix glycoprotein that is ubiquitously expressed by malignant gliomas and probably contributes to tumor cell adhesion, invasion, migration and proliferation. For tenascin-C inhibition, RNA interference intervention (iRNAi) approach have been applied.03-25-2010
20130158101REGULATORS OF NFAT AND/OR STORE-OPERATED CALCIUM ENTRY - Embodiments of the inventions relate to modulating NFAT activity, modulating store-operated Ca06-20-2013
20130158100INTRACELLULAR DNA RECEPTOR - Provided herein are methods of identifying and using compounds that modulate an AIM2 polypeptide-mediated immune response. Further provided herein are methods of treating disease comprising administering to a patient a compound that decreases expression of an AIM2 polypeptide. Further provided herein are methods of providing gene therapy to a patient comprising administering to the patient a gene therapy agent and a compound that decreases expression of an AIM2 polypeptide. In certain embodiments, a compound that decreases expression of an AIM2 polypeptide comprises an siRNA or an shRNA.06-20-2013
20120245217RNAi-MEDIATED INHIBITION OF HIF1A FOR TREATMENT OF OCULAR ANGIOGENESIS - RNA interference is provided for inhibition of HIF1A mRNA expression for treating patients with ocular angiogenesis, particularly for treating retinal edema, diabetic retinopathy, sequela associated with retinal ischemia, posterior segment neovascularization (PSNV), and neovascular glaucoma, and for treating patients at risk of developing such conditions.09-27-2012
20120245216COMPOSITIONS AND METHODS FOR TREATMENT OF NEUROPATHIC PAIN - The present invention relates to compounds, compositions, methods, systems and kits for treating neuropathic pain regulated by SIP30. The present invention provides SIP30 antagonists for the treatment of neuropathic pain.09-27-2012
20130158099Methods for Identifying Agents that Inhibit Cell Migration, Promote Cell Adhesion and Prevent Metastasis - Disclosed are methods for identification of agents that modulate cell attachment, cell migration and cell viability. Cancer and primary cells adhered to a matrix are treated with agent(s) that modulate ActRII signaling and cell adhesion. Agents are tested that modulate cell adhesion, detachment, invasion and viability. Agents that modulate the expression, phosphorylation, function and translocation of ActRII signaling pathway members also can predict agents that modulate cell adhesion, detachment, invasion and viability. The methods have utility in identifying agents that prevent cancer cell metastasis, wound dehiscence, aortic dissection and aid retina attachment and skin replacement and fertility.06-20-2013
20130158098Alkenyl Substituted Cycloaliphatic Compounds as Chemical Inducers of Proximity - Methods of inducing proximity of chimeric molecules in a cell are provided. Aspects of the methods include contacting a cell with an amount of alkenyl substituted cycloaliphatic (ASC) inducer compound, e.g., abscisic acid, effective to induce proximity of first and second chimeric molecules. Also provided are compositions and kits for practicing various embodiments of the methods. Methods of the invention find use in a variety of different applications, including transcription induction applications.06-20-2013
20130158097COMPOSITIONS AND METHODS DIRECTED TO TREATING LIVER FIBROSIS - The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the COL1A1, TGF-β, and SMAD2/3 genes, and methods of using such dsRNA compositions to inhibit expression of COL1A1, TGF-β, and SMAD2/3.06-20-2013
20130158096MIRNAS INVOLVED IN THE BLOOD BRAIN BARRIER FUNCTION - The current invention relates to use of particular nucleic acids for modulating (increasing or decreasing) blood-brain barrier function, and for use of such nucleic acids in treatment of conditions involving blood brain barrier function, including multiple sclerosis, HIV infection, Alzheimer's disease, Parkinson's disease, epilepsy, and so on. Also provided is an assay for identifying drugs useful in modulating blood brain barrier function.06-20-2013
20130079383Lipid Compounds Targeting VLA-4 - The invention relates to the compounds of formula I:03-28-2013
20130030039NOVEL MULTIDRUG RESISTANCE-ASSOCIATED POLYPEPTIDE - Compositions and methods are disclosed for improving the effectiveness of a chemotherapeutic regimen to eradicate multidrug-resistant transformed cells from the body of a mammal, preferably from the body of a human. The present disclosure capitalizes on the discovery of a novel multidrug-resistance associated protein (MRP), herein designated MRP-β. The disclosed compositions include MRP-β nucleic acids, including probes and antisense oligonucleotides, MRP-β polypeptides and antibodies, MRP-β expressing host cells, and non-human mammals transgenic or nullizygous for MRP-β. The disclosed methods include methods for attenuating aberrant MRP-β gene expression, protein production and/or protein function. In addition, methods are disclosed for identifying and using a modulator, such as an inhibitor, of MRP-β. Preferably, the modulator is a small molecule.01-31-2013
20130030038Dermatological, Pharmaceutical Composition Suitable for Oligonucleotides - The invention relates to a cosmetic and/or dermatological and/or pharmaceutical composition for the topical use and application of oligonucleotides, in particular antisense-oligonucleotides such as DNAzyme, siRNAs, asDNAs or ribozymes for use as an agent against inflammatory diseases by means of emulsions having a dispersed, internal, discontinuous aqueous phase.01-31-2013
20130030037RNAi Modulation Of RSV And Therapeutic Uses Thereof - The present invention is based on the in vivo demonstration that RSV can be inhibited through intranasal administration of iRNA agents as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the present invention provides general and specific compositions and methods that are useful in reducing RSV mRNA levels, RSV protein levels and viral titers in a subject, e.g., a mammal, such as a human. These findings can be applied to other respiratory viruses.01-31-2013
20130030036MODULATION OF FORKHEAD BOX O1A EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of forkhead box O1A. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding forkhead box O1A. Methods of using these compounds for modulation of forkhead box O1A expression and for treatment of diseases associated with expression of forkhead box O1A are provided, in particular, for methods of treating diabetes.01-31-2013
20130030035IDENTIFICATION OF MICRO-RNAS INVOLVED IN NEUROMUSCULAR SYNAPSE MAINTENANCE AND REGENERATION - The present invention relates to the identification of miRNAs that are involved in the process of neuromuscular synaptic maintenance and regeneration following injury or disease. Modulation of these miRNAs is proposed as treatment for spinal cord injury and neurodegenerative disease.01-31-2013
20130030034MODULATION OF TIMP1 AND TIMP2 EXPRESSION - Provided herein are compositions, methods and kits for modulating expression of target genes, particularly of tissue inhibitor of metalloproteinase 1 and of tissue inhibitor of metalloproteinase 2 (TIMP1 and TIMP2, respectively). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding TIMP1 and TIMP2, for example, the gene encoding human TIMP1 and TIMP2. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with TIMP1 and TIMP2 including fibrotic diseases and disorders including liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis.01-31-2013
20130079389siRNA And Their Use In Methods And Compositions For The Treatment And/Or Prevention Of Eye Conditions - The invention relates to methods and compositions for the treatment/and or prevention of eye conditions related to high levels of expression and/or activity of the vanilloid-1 receptor (TRPV).03-28-2013
20130079386DIAGNOSTICS OF B-CELL LYMPHOMA - The present invention relates to the fields of genetics and oncology and provides methods and means for diagnosing and monitoring of patients having B-cell lymphomas, such methods and means allowing an early diagnosis of the B-cell lymphoma. Specifically, the present invention relates to a novel method and a biomarker for diagnosing B-cell lymphomas and for differentiating the B-cell lymphomas into prognostic groups of indolent and aggressive B-cell lymphomas.03-28-2013
20130079388FORMULATIONS COMPRISING ANTISENSE NUCLEOTIDES TO CONNEXINS - A therapeutic and/or cosmetic formulation comprising at least one anti-sense polynucleotide to a connexin protein together with a pharmaceutically acceptable carrier or vehicle is useful in site specific down regulation of connexin protein expression, particularly in reduction of neuronal cells death, wound healing, reduction of inflammation, decrease of scar formation and skin rejuvenation and thickening.03-28-2013
20130079384Means and Methods for Determining Risk of Cardiovascular Disease - The invention relates to medicine, in particular to internal medicine and/or cardiology. The present invention provides means and methods for typing a sample and identifying and/or treating a patient suffering from or at risk of suffering from cardiovascular disease by measuring mi RNA present in a sample of said patient. The present invention further provides means and methods for identifying new cardiovascular disease therapies.03-28-2013
20130079385USP47 Inhibtors and Methods to Induce Apoptosis - The present invention relates to USP47 (ubiquitin specific protease 47) inhibitors and methods for inducing apoptosis or cell death in a target cell. In certain embodiments, the invention relates to methods and kits to screen for related agents that induce apoptosis. Additionally, the invention relates to assays for screening compounds capable of acting as USP47 inhibitors.03-28-2013
20130079382Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro - Compositions and methods for down modulating target gene expression with RNA interference, as well as methods for administering said compositions are disclosed. The method comprises administering a first strand to a cell, incubating the cell for a time period suitable for uptake of the first oligo prior to administering a second strand, wherein the first strand and said second strand form an intracellular duplex which is effective to catalyze degradation of gene target mRNA or inhibit translation of said mRNA.03-28-2013
20130158095EML4-ALK FUSION GENE - The present inventors found that a fusion gene present in some cancer patients is an oncogene. The present invention relates to a polypeptide as a novel fusion protein, a polynucleotide encoding the polypeptide, a vector comprising the polynucleotide, a transformed cell comprising the vector, a method for detecting the fusion protein or polynucleotide, a method for screening a therapeutic agent for cancer, and a method for treating cancer that is shown to be positive for the fusion gene. Further, the present invention relates kit, primer set, and probe useful in the detection of cancer that is shown to be positive for the fusion gene.06-20-2013
20130035368OLIGONUCLEOTIDE COMPOUNDS COMPRISING NON-NUCLEOTIDE OVERHANGS - The invention relates to siRNA compounds comprising one non-nucleotide moiety covalently attached to at least one of the sense or antisense strands to down-regulate the expression of human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier and to methods of treating and/or preventing the incidence or severity of various diseases or conditions associated with the target genes and/or symptoms associated with such diseases or conditions.02-07-2013
20130035373TREATMENT OF FIBROBLAST GROWTH FACTOR 21 (FGF21) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO FGF21 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Fibroblast growth factor 21 (FGF21), in particular, by targeting natural antisense polynucleotides of Fibroblast growth factor 21 (FGF21). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of FGF21.02-07-2013
20130035372TREATMENT OF COLONY-STIMULATING FACTOR 3 (CSF3) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO CSF3 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Colony-stimulating factor 3 (CSF3), in particular, by targeting natural antisense polynucleotides of Colony-stimulating factor 3 (CSF3). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of CSF3.02-07-2013
20130035371LIPID FORMULATED DSRNA TARGETING THE PCSK9 GENE - This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene.02-07-2013
20130035369DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION - The present invention provides double stranded compositions wherein the first strand is modified to have a particular motif and the second strand is modified a selected motif. More particularly, the present compositions comprise an antisense strand that is modified to have a positional/full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional/full motif or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes.02-07-2013
20130035370COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION - Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.02-07-2013
20130035367ANTISENSE OLIGONUCLEOTIDES THAT TARGET A CRYPTIC SPLICE SITE IN USH1C AS A THERAPEUTIC FOR USHER SYNDROME - The present invention provides a method for treating Usher's syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript.02-07-2013
20130035365Anti-sense oligonucleotides targeted against exon 9 of IL-23R alpha gene and method of using same to induce exon skipping and to treat inflammatory bowel diseases - The present invention relates to anti-sense oligonucleotides (AONs) used to induce exon 9 skipping in IL-23Rα gene. Exon 9 skipping of the IL23Rα gene ultimately causes specific induction of a novel soluble truncated IL-23Rα (Δ9) protein, characterized by a lack in a transmembrane domain and has a unique eight (8) amino acids (GLKEGSYC) at its C-terminus end as a result of frame-shift. The present invention provides a utility application of the use of AONs to induce production of a Δ9 protein which inhibits IL-23R-mediated cell signaling. More particularly, Δ9 protein blocks STAT3 formation as well as Th17 maturation. There is provided a therapeutic application of AONs in treating a mammal such as a human patient inflicted with Crohn's disease.02-07-2013
20130035374MicroRNA induction of pluripotential stem cells and uses thereof - Compositions and methods for inducing the formation of an induced pluripotential stem (iPS) cell from a somatic cell are disclosed. The compositions comprise miR 302-367 cluster and valproic acid. Further disclosed are methods for treatment of a disease or condition in a subject through the use of the compositions.02-07-2013
20130035366MODULATION OF HEPATITIS B VIRUS (HBV) EXPRESSION - Disclosed herein are antisense compounds and methods for decreasing HBV mRNA, DNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate HBV-related diseases, disorders or conditions.02-07-2013
20130041017SIRNA-BASED THERAPY OF FIBRODYPLASIA OSSIFICANS PROGRESSIVA (FOP) - This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to compositions and methods for siRNA-based regulation of mutated ACVR1 expression in the treatment of Fibrodysplasia Ossificans Progressiva (FOP).02-14-2013
20130041016Compositions and Methods for Preventing and Treating Cancer via Modulating UBE1L, ISG215 and/or UBP43 - Compositions and methods of using compositions that induce UBE1L or a ubiquitin-like protein ISG15, or inhibit a deconjugase UBP43 to degrade oncogenic proteins and enhance apoptosis of cancer (neoplastic) or pre-cancerous (pre-neoplastic) cells are provided. Methods for the prevention or treatment of cancer via administration of these compositions are also provided.02-14-2013
20130041015Use of the Lactosylceramide Synthase Isoform B1,4GALT-V as a Biomarker for Cancer - In one aspect, B1,4GalT-V, an isoform of the enzyme lactosylceramide synthase, is provided as a biomarker for cancer. Also provided are methods and compositions directed at cancers characterized by the overexpression or upregulation of the lactosylceramide synthase isoform B1,4GalT-V.02-14-2013
20130041014ISOFORM OF BRUTON'S TYROSINE KINASE (BTK) PROTEIN - The use of compounds is described which are capable of functionally blocking at least one of the genes chosen from the group composed of EphAl, EphA2, EphA8, EphB2, CSF1R, VEGFR2, RAMP2, RAMP3, CLRN1, MAPK4, PIK3C2A, PIK3CG, GSK3alpha, GSK3beta, IRAK3, DAPK1, JAK1, PIM1, TRB3, BTG1, LATS1, LIMK2, MYLK, PAK1, PAK2, CDC2, BTK, PNRC2, NCOA4, NR2C1, TPR, RBBP8, TRPC7, FXYD1, ERNI, PRSS16, RPS3, CCL23 and SERPINE1, for the manufacture of a medicament destined to diminish the resistance to chemotherapeutic drugs in the therapeutic treatment of epithelial tumour pathologies. Also described is a method for the determination of the drug resistance in tumour cells, as well as a method for the identification of tumour stem cells.02-14-2013
20130041013ISOFORM OF BRUTON'S TYROSINE KINASE (BTK) PROTEIN - The use of compounds is described which are capable of functionally blocking at least one of the genes chosen from the group composed of EphA1, EphA2, EphA8, EphB2, CSF1R, VEGFR2, RAMP2, RAMP3, CLRN1, MAPK4, PIK3C2A, PIK3CG, GSK3alpha, GSK3beta, IRAK3, DAPK1, JAK1, PIM1, TRB3, BTG1, LATS1, LIMK2, MYLK, PAK1, PAK2, CDC2, BTK, PNRC2, NCOA4, NR2C1, TPR, RBBP8, TRPC7, FXYD1, ERNI, PRSS16, RPS3, CCL23 and SERPINE1, for the manufacture of a medicament destined to diminish the resistance to chemotherapeutic drugs in the therapeutic treatment of epithelial tumour pathologies. Also described is a method for the determination of the drug resistance in tumour cells, as well as a method for the identification of tumour stem cells.02-14-2013
20130041012NEW ISOFORM OF BRUTON'S TYROSINE KINASE (BTK) PROTEIN - The use of compounds is described which are capable of functionally blocking at least one of the genes chosen from the group composed of EphAI, EphA2, EphA8, EphB2, CSF1R, VEGFR2, RAMP2, RAMP3, CLRN1, MAPK4, PIK3C2A, PIK3CG, GSK3alpha, GSK3beta, IRAK3, DAPK1, JAK1, PIM1, TRB3, BTG1, LATS1, LIMK2, MYLK, PAK1, PAK2, CDC2, BTK, PNRC2, NCOA4, NR2C1, TPR, RBBP8, TRPC7, FXYD1, ERNI, PRSS16, RPS3, CCL23 and SERPINE1, for the manufacture of a medicament destined to diminish the resistance to chemotherapeutic drugs in the therapeutic treatment of epithelial tumour pathologies. Also described is a method for the determination of the drug resistance in tumour cells, as well as a method for the identification of tumour stem cells.02-14-2013
20130041011BASE MODIFIED BICYCLIC NUCLEOSIDES AND OLIGOMERIC COMPOUNDS PREPARED THEREFROM - Provided herein are novel base modified bicyclic nucleosides, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, novel pyrimidine bicyclic nucleosides are provided wherein each pyrimidine base is substituted at the 5 position with an optionally substituted, aromatic or heteroaromatic ring system comprising from 5 to 7 ring atoms selected from C, N, O and S. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.02-14-2013
20130041010COMPOSITIONS AND METHODS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DSRNA CONTAINING MODIFIED NUCLEOTIDES - The invention features compositions and methods that are useful for reducing the expression or activity of a specified gene in eukaryotic cell.02-14-2013
20130041009Methods and means for increasing resistance to cell damage - Methods are provided to increase resistance to cell damage in a subject. The increase in resistance to cell damage in a subject in the subject is accomplished by decreasing activity of eEF2 kinase in the subject. The eEF2 kinase activity can be decreased by decreasing the amount of functional eEF2 kinase produced by the subject, including contacting the eEF2 kinase with a compound that inhibits phosphorylation of eEF2 kinase substrate or decreasing the amount of functional eEF2 kinase is decreased by reducing expression of a gene encoding the eEF2 kinase.02-14-2013
20130079387ANTISENSE OLIGONUCLEOTIDE MODULATION OF RAF GENE EXPRESSION - Oligonucleotides are provided which are targeted to nucleic acids encoding human raf and capable of inhibiting raf expression. The oligonucleotides may have chemical modifications at one or more positions and may be chimeric oligonucleotides. Methods of inhibiting the expression of human raf using oligonucleotides of the invention are also provided. The present invention further comprises methods of inhibiting hyperproliferation of cells and methods of treating or preventing conditions, including hyperproliferative conditions, associated with raf expression.03-28-2013
20090275632Methods of diagnosis and treatment - A method of diagnosing a disease in which an miRNA is expressed at a lower level comprises, in a test sample obtained from a subject, determining the methylation status of at least one gene encoding the miRNA in the sample, wherein the presence of methylation is indicative of the presence of the disease. Methylation of the gene causes a down regulation in expression which may also be monitored. Related methods and kits are also described based upon methylation of miRNA encoding genes. The primary miRNA of interest is 124a miRNA.11-05-2009
20090247605Treating diseases mediated by metalloprotease-shed proteins - This invention relates to the identification of membrane-associated proteins shed by metalloproteinases and in particular by TNF-alpha converting enzyme (TACE), to the use of such metalloproteinase-shed proteins in assays for TACE agonists and antagonists, and to the use of metalloproteinase agonists and antagonists, and particularly TACE agonists and antagonists, in the treatment of diseases mediated by certain shed proteins.10-01-2009
20090258923Carrier Composition for Nucleic Acid Transport - An object of the present invention is to provide a nucleic acid delivery carrier composition of low toxicity and high safety, the carrier composition, when used to administer a nucleic acid such as an siRNA into an animal-derived cell or organism, being capable of delivering efficiently the nucleic acids into the cells while protecting it from being degraded; and a nucleic acid deliver composition containing the carrier and a nucleic acid.10-15-2009
20090258928METHODS AND COMPOSITIONS FOR DIAGNOSING AND MODULATING HUMAN PAPILLOMAVIRUS (HPV) - The present invention concerns methods and compositions for treating a patient having, suspected of having, or at risk of obtaining a HPV infection.10-15-2009
20110263680REDUCED SIZE SELF-DELIVERING RNAI COMPOUNDS - The present invention relates to RNAi constructs with minimal double-stranded regions, and their use in gene silencing. RNAi constructs associated with the invention include a double stranded region of 8-14 nucleotides and a variety of chemical modifications, and are highly effective in gene silencing. The RNAi constructs may be, for instance, miRNA constructs that are miRNA modulators.10-27-2011
20100099744RNA INTERFERENCE MEDIATED INHIBITION OF MATRIX METALLOPROTEINASE 13 (MMP13) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating matrix metalloproteinase (e.g., MMP13) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of MMP13 gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of MMP13 genes.04-22-2010
20100099738POLYETHYLENE GLYCOL LIPID CONJUGATES AND USES THEREOF - Polyethylene glycol (PEG)-lipid conjugates, polyethylene glycol (PEG)-lipid conjugate based drug delivery systems, ways to make them and methods of treating diseases using them are disclosed.04-22-2010
20130210887DENDRIMERS AS NON-VIRAL VEHICLES FOR GENE THERAPY - The present invention relates to novel compounds of general formula (I) and (II) for their use in gene therapy as non-viral vehicles and their use for the preparation of a medicament. It also discloses the process of synthesis of said compounds of general formula (I) and (II).08-15-2013
20120264812TREATMENT OF NUCLEAR RESPIRATORY FACTOR 1 (NRF1) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO NRF1 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Nuclear Respiratory Factor 1 (NRF1), in particular, by targeting natural antisense polynucleotides of Nuclear Respiratory Factor 1 (NRF1). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of NRF1.10-18-2012
20120264811Elongator Proteins and Use Thereof as DNA Demethylases - The invention provides DNA demethylases comprising Elp1, Elp2, Elp3, Elp4, Elp5 and/or Elp6. The invention also provides methods of modulating gene expression, for example, for the treatment of cancer or to modify the cellular transcription program (e.g., for regenerative medicine). Also provided are methods of identifying compounds that modulate the DNA demethylase activity of the DNA demethylases of the invention.10-18-2012
20120264810COMPOSITIONS AND METHODS FOR ENHANCING CELLULAR UPTAKE AND INTRACELLULAR DELIVERY OF LIPID PARTICLES - Compositions, methods and compounds useful for enhancing the uptake of a lipid particle b\ a cell are described In particular embodiments, the methods of the invention include contacting a cell with a lipid particle and a compound that binds a Na+/K+ ATPase to enhance uptake of the lipid particle b\ the cell Related compositions useful in practicing methods include lipid particles comprising a conjugated compound that enhances uptake of the lipid particles b\ the cell The methods and compositions are useful in delivering a therapeutic agent to a cell, e g for the treatment of a disease or disorder in a subject10-18-2012
20130137750DOUBLE STRANDED RNA COMPOUNDS TO RHOA AND USE THEREOF - The present invention relates to compounds, pharmaceutical compositions comprising same, methods of use thereof and kits for the down-regulation of RhoA gene. The compounds, compositions, methods and kits are useful in the treatment of subjects suffering from diseases or conditions and or symptoms associated with diseases or conditions in which RhoA expression has adverse consequences and for conferring neuroprotection.05-30-2013
20100144837DIAGNOSIS AND TREATMENT OF MALIGNANT NEOPLASMS - The invention features a method for diagnosing a malignant neoplasm in a mammal by contacting a bodily fluid from the mammal with an antibody which binds to an human aspartyl (asparaginyl) beta-hydroxylase (HAAH) polypeptide and methods of treating malignant neoplasms by inhibiting HAAH.06-10-2010
20100144834METHODS FOR TREATING HYPERCHOLESTEROLEMIA - Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating hypercholesterolemia, or alternatively for treating or preventing atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.06-10-2010
20100144839ANTI-CANCER OLIGODEOXYNUCLEOTIDES - It is disclosed herein that suppressive ODNs are of use for preventing or delaying the formation of a tumor, reducing the risk of developing a tumor, treating a tumor, preventing conversion of a benign to a malignant lesion, or preventing metastasis. In some embodiments, methods are disclosed herein for treating, preventing or reducing the risk of developing a tumor, such as esophageal, gastrointestinal, liver, lung, skin and colon tumors or a mesothelioma. Generally, the methods disclosed herein include selecting a subject for treatment and administering to the subject a therapeutically effective amount of one or more suppressive ODN. In some examples, additional agents can also be administered to the subject of interest.06-10-2010
20100144841CONTROL OF NK CELL FUNCTION AND SURVIVAL BY MODULATION OF SHIP ACTIVITY - Inhibition of dendritic cell function in solid organ grafts or allogeneic bone marrow transplants prior to or during engraftment by blocking SH2-containing inositol phosphatase (SHIP) expression or function is taught as a method of abrogating immune rejection and thereby increasing the efficacy of engraftment of an allogeneic bone marrow transplant or solid organ allograft or xenograft. Also disclosed is a transgenic mouse having the genotype SHIP06-10-2010
20100144832Prostate Cancer-Specific Alternations in ERG Gene Expression and Detection and Treatment Methods Based on Those Alterations - The disclosure describes alterations in ERG gene expression. ERG isoforms and promoter sequence of the ERG gene that are involved in, or associated with, prostate cancer are provided. The disclosure further provides therapeutic compositions and methods of detecting, diagnosing, prognosing, and treating prostate cancer, including biomarkers for detecting the expression of two or more of the following genes: PSA/KLK3, PMEPA1, NKX3.1, ODC1, AMD1, and ERG.06-10-2010
20100324120LIPID FORMULATION - The invention features a cationic lipid of formula I,12-23-2010
20100041735MODULATION OF TOLL-LIKE RECEPTOR 3 EXPRESSION BY ANTISENSE OLIGONUCLEOTIDES - Antisense oligonucleotide compounds, compositions and methods are provided for down regulating the expression of TLR3. The compositions comprise antisense oligonucleotides targeted to nucleic acids encoding TLR3. The compositions may also comprise antisense oligonucleotides targeted to nucleic acids encoding TLR3 in combination with other therapeutic and/or prophylactic compounds and/or compositions. Methods of using these compounds and compositions for down-regulating TLR3 expression and for prevention or treatment of diseases wherein modulation of TLR3 expression would be beneficial are provided.02-18-2010
20100041736Isolated MCPIP and Methods of Use - A monocyte chemoattractant protein (MCP-1)-inducible protein, MCPIP, its polynucleotide and amino acid sequences from mouse and human, and methods for its use are disclosed.02-18-2010
20100041734MODULATION OF TOLL-LIKE RECEPTOR 7 EXPRESSION BY ANTISENSE OLIGONUCLEOTIDES - Antisense oligonucleotide compounds, compositions and methods are provided for down regulating the expression of TLR7. The compositions comprise antisense oligonucleotides targeted to nucleic acids encoding TLR7. The compositions may also comprise antisense oligonucleotides targeted to nucleic acids encoding TLR7 in combination with other therapeutic and/or prophylactic compounds and/or compositions. Methods of using these compounds and compositions for down-regulating TLR7 expression and for prevention or treatment of diseases wherein modulation of TLR7 expression would be beneficial are provided.02-18-2010
20100041733NOVEL RNAi THERAPEUTIC FOR TREATMENT OF HEPATITIS C INFECTION - Small interfering RNAs (siRNAs) or small hairpin RNA (shRNAs) and compositions comprising same are provided that specifically target human cyclophilin A (CyPA) to effectively inhibit Hepatitis C(HCV) infection in a cell. Such siRNA and shRNAs may have a length of from about 19 to about 29 contiguous nucleotides corresponding to a specific region of human cyclophilin A (CyPA) cDNA of from about nucleotide 155 to about nucleotide 183 having particular potency against CyPA and HCV. Such siRNA and shRNAs may be formulated as naked compositions or as pharmaceutical compositions. DNA polynucleotides, plasmids, and viral or non-viral vectors are also provided that encode siRNA or shRNA molecules, which may be delivered directly to cells or in combination with known delivery agents, such as lipids, polymers, encapsulated lipid particles, such as liposomes. Methods for treating, managing inhibiting, preventing, etc., HCV infection using such siRNA and shRNAs and compositions comprising same are also provided.02-18-2010
20100041732METHODS FOR IDENTIFYING COMPOUNDS THAT INHIBIT HIV INFECTION - This invention provides novel methods for identifying agents that inhibit HIV infection. The anti-HIV agents are identified by screening test compounds for ability to down-regulate the kinase activity or the expression of a PAK3 molecule. Such PAK3 modulators can be further examined for their activity in inhibiting HIV infection. These novel anti-HIV agents are useful in the prevention or treatment of HIV infection and conditions associated with HIV infection.02-18-2010
20100249213MicroRNA Signatures in Human Ovarian Cancer - The present invention provides novel methods and compositions for the diagnosis, prognosis and treatment of ovarian cancer. The invention also provides methods of identifying anti-cancer agents.09-30-2010
20100022618Long interfering nucleic acid duplexes targeting multiple RNA targets - Long interfering nucleic acid (iNA) duplexes, which are at least 30 nucleotides in length, which have at least one nick or nucleotide gap in the antisense or the sense strands or in both the sense and antisense strands. These long iNA duplexes do not induce an interferon response when transfected into mammalian cells. The antisense strands can target two separate mRNAs or two segments of one mRNA.01-28-2010
20100105760Treatment of Apolipoprotein-A1 Related Diseases by Inhibition of Natural Antisense Transcript to Apolipoprotein-A1 - Oligonucleotide compounds modulate expression and/or function of an apolipoprotein (ApoA1) polynucleotides and encoded products thereof. Methods for treating diseases associated with apolipoprotein-A1 (ApoA1) comprise administering one or more Oligonucleotide compounds designed to inhibit the Apo-A1 natural antisense transcript to patients.04-29-2010
20100105759H19 SILENCING NUCLEIC ACID AGENTS FOR TREATING RHEUMATOID ARTHRITIS - The invention relates to the treatment of rheumatoid arthritis, particularly to the use of nucleic acid agents capable of silencing H19 for the treatment of rheumatoid arthritis. The invention provides methods for ameliorating rheumatoid arthritis and symptoms associated therewith, utilizing gene silencing oligonucleotides such as small interfering RNA (siRNA) agents directed to H19.04-29-2010
20100105758USE OF AN ADENOSINE ANTAGONIST - Uses for a selective adenosine A3 receptor antagonist, or RNAi directed against said receptor, to treat myocardial infarction and heart conditions including heart failure, are provided. Optionally, an adenosine A2a receptor agonist may also be used with the adenosine A3 receptor antagonist. Methods of treating heart failure are also provided.04-29-2010
20100076059METHOD OF EVALUATING COMPOUND EFFICACIOUS IN TREATING OBESITY BY USING Slc25a10 - Evaluation of compounds including screening of therapeutic agents for obesity is performed utilizing expression levels of S1c25a10 gene or protein in a test tissue or a test cell, or utilizing the nature of S1c25a10 gene or protein. Examination of obesity is performed based on expression levels of S1c25a10 gene or polymorphisms of the gene.03-25-2010
20100093832MODULATION OF INSULIN LIKE GROWTH FACTOR I RECEPTOR EXPRESSION IN CANCER - Provided herein are methods, compounds, and compositions for reducing expression of an IGF-IR mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions that inhibit expression of IGF-IR in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate the tumor or cancer, or a symptom thereof.04-15-2010
20100093833Methods for Inducing Differentiation of Undifferentiated Mammalian Cells Into Osteoblasts - The present invention relates to in vivo and in vitro methods, agents and compound screening assays for inducing differentiation of undifferentiated mammalian cells into osteoblasts, including bone formation enhancing pharmaceutical compositions, and the use thereof in treating and/or preventing a disease involving a systemic or local decrease in mean bone density in a subject.04-15-2010
20120165398PHARMACEUTICAL COMPOSITIONS AND METHODS USEFUL FOR MODULATING ANGIOGENESIS, INHIBITING METASTASIS AND TUMOR FIBROSIS, AND ASSESSING THE MALIGNANCY OF COLON CANCER TUMORS - Methods and compositions suitable for modulating angiogenesis in a mammalian tissue are provided. Further provided are methods suitable for inhibiting metastasis and fibrosis in a mammalian tissue and for assessing the malignancy of colon cancer tumors.06-28-2012
20120165396MODULATION OF EXPORTIN 5 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of exportin 5. The compositions comprise oligonucleotides, targeted to nucleic acid encoding exportin 5. Methods of using these compounds for modulation of exportin 5 expression and for diagnosis and treatment of diseases and conditions associated with expression of exportin 5 are provided.06-28-2012
20120165387General composition framework for ligand-controlled RNA regulatory systems - The invention provides an improved design for the construction of extensible nucleic acid-based, ligand-controlled regulatory systems, and the nucleic acid regulatory systems resulting therefrom. The invention contemplates improving the design of the switches (ligand-controlled regulatory systems) through the design of an information transmission domain (ITD). The improved ITD eliminates free-floating ends of the switching and the competing strands, and localizes competitive hybridization events to a contiguous strand of competing and switching strands in a strand-displacement mechanism-based switch, thereby improving the kinetics of strand-displacement. The improved regulatory systems have many uses in various biological systems, including gene expression control or ligand-concentration sensing.06-28-2012
20100069462MITOCHONDRIAL FUNCTION OF PROHIBITIN 2 (PHB2) - The present invention relates to a PHB2 gene regulator and a therapeutic drug for mitochondrial-function-related disease containing the same, for example.03-18-2010
20130046009RHO KINASE INHIBITORS FOR TREATMENT OF MASTOCYTOSIS AND ACUTE MYELOID LEUKEMIA - The disclosure is directed to methods of treating hematologic malignancies. More particularly, the disclosure is directed to methods of treating hematologic malignancies using Rho kinase (ROCK) inhibitors and myosin light chain-specific inhibitory RNA molecules. The disclosure is further directed to methods of identifying drug candidates for inhibiting ROCK in hematologic malignancies.02-21-2013
20130046010SUMO AS A MARKER OF CANCER DEVELOPMENT AND TARGET FOR CANCER THERAPY - Disclosed herein are methods relating to inhibiting or reducing proliferation of a cancer cell, for treating cancer in a subject in need of treatment, predicting the risk of progression of cancer to a more aggressive cancer, and screening for cancer in a subject, that comprise detecting and/or decreasing the levels of SUMO conjugated proteins and detecting and/or interfering with SUMO conjugation.02-21-2013
20130046008SELECTIVE REDUCTION OF ALLELIC VARIANTS - Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).02-21-2013
20130046007SELECTIVE REDUCTION OF ALLELIC VARIANTS - Disclosed herein are antisense compounds and methods for selectively of reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative, such as Huntington's Disease (HD).02-21-2013
20130090366MODULATION OF EPIDERMAL GROWTH FACTOR RECEPTOR LIGANDS - The present invention relates to a method for modulating the expression and/or activity of an epidermal growth factor receptor (EGFR) ligand in a cell or tissue, the method comprising contacting the cell or tissue with a miR-7 miRNA, a pre-cursor or variant thereof, a miRNA comprising a seed region comprising the sequence GGAAGA, or an antagonist of any such miRNA.04-11-2013
20110003879Antisense oligonucleotides targeted to the coding region of thymidylate synthase and uses thereof - Antisense oligonucleotides directed to the coding region of a mammalian thymidylate synthase mRNA that are capable of inhibiting the proliferation of cancer cells without decreasing the level of thymidylate synthase mRNA in the cells are provided. The antisense oligonucleotides are also capable of inducing apoptosis in the cancer cells. The antisense oligonucleotides can be used to inhibit the proliferation of cancer cells and to induce apoptosis in cancer cells. Methods of treating cancer, and in particular breast cancer, with the antisense oligonucleotides, alone or in combination with other therapeutics, are also provided.01-06-2011
20130090369TRANSFECTION SHEETS AND METHODS OF USE - The present disclosure relates to nucleic acid-lipid compositions for use in delivering nucleic acids to cells in vitro or in vivo. In particular, it relates to the preparation and use of resilient transfection sheets that comprise the nucleic acid-lipid compositions.04-11-2013
20130090373AGENT FOR SUPPRESSING EXPRESSION OF DOMINANT ALLELE - An agent for selectively suppressing the expression of a dominant allele while allowing expression of wild-type or desired alleles and methods for using the agent are described. The RNAi agent has a structure obtained by assigning a dominant point mutation in the targeted allele as a standard point, setting a base length from the standard point to the 5′ end to a predetermined length, and introducing one mismatch base differing from the target sequence to a predetermined position downstream from the standard point.04-11-2013
20130090371METHODS AND COMPOSITIONS FOR INHIBITION OF BETA2-ADRENERGIC RECEPTOR DEGRADATION - The present invention generally relates to compositions and kits comprising a β2-AR agonist and a modulator of a β2-AR regulator gene, where the modulator of the β2-AR regulator gene inhibits the internalization and/or degradation of the β2-ad-renergic receptor (β2-AR). More specifically, the present invention relates to the use of an agonist of β2-adrenergic receptor (β2-AR) and an agent which inhibits agonist induced β2-adrenergic receptor (β2-AR) internalization and/or degradation in method for the treatment of a respiratory disorder in a subject.04-11-2013
20130090372Novel Low Molecular Weight Cationic Lipids for Oligonucleotide Delivery - The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.04-11-2013
20130090370APOPTOSIS INDUCER FOR CANCER CELL - The present invention revealed that by suppressing the expression of the WRN gene, the BLM gene, or the RecQ1 gene, which belong to the RecQ helicase family, apoptosis is induced in various cancer cells and their proliferation is suppressed. Compounds that suppress the expression of RecQ helicase family genes or the functions of RecQ helicase proteins are thought to have the activity of inducing apoptosis.04-11-2013
20130090368THERAPEUTIC ANTISENSE OLIGONUCLEOTIDE COMPOSITIONS FOR THE TREATMENT OF INFLAMMATORY BOWEL DISEASE - Disclosed herein is a method for the sustained amelioration and/or treatment of ulcerative colitis comprising rectal administration of a compound comprising an antisense oligonucleotide having the sequence 5′-GCCCAAGCTGGCATCCGTCA-3′, ISIS 2302. The method results in a decrease in the indications of ulcerative colitis for an extended period (greater than 90 days) after the conclusion of the administration of the composition. The composition is well tolerated and systemic exposure is minimal.04-11-2013
20100004318METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene.01-07-2010
20090326046METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.12-31-2009
20090318533Annexin A9 (ANXA9) Biomarker and Therapeutic Target in Epithelial Cancer - Amplification of the ANXA9 gene in human chromosomal region 1q21 in epithelial cancers indicates a likelihood of both in vivo drug resistance and metastasis, and serves as a biomarker indicating these aspects of the disease. ANXA9 can also serve as a therapeutic target. Interfering RNAs (iRNAs) (such as siRNA and miRNA) and shRNA adapted to inhibit ANXA9 expression, when formulated in a therapeutic composition, and delivered to cells of the tumor, function to treat the epithelial cancer.12-24-2009
20090312397RNAi Modulation Of ApoB And Uses Thereof - The invention relates to compositions and methods for modulating the expression of apolipoprotein B, and more particularly to the downregulation of apolipoprotein B by chemically modified oligonucleotides.12-17-2009
20090312396METHODS FOR CANCER TREATMENT USING TAK1 INHIBITORS - The invention includes, in part, a method of inhibiting lymphoid tumour cell proliferation by contacting the lymphoid with a TAK1 inhibitor.12-17-2009
20100267806LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 AND VEGF GENES - This invention relates to compositions containing double-stranded ribonucleic acid (dsRNA) in a lipid formulation, and methods of using the compositions to inhibit the expression of the Human kinesin family member 11 (Eg5) and Vascular Endothelial Growth Factor (VEGF), and methods of using the compositions to treat pathological processes mediated by Eg5 and VEGF expression, such as cancer.10-21-2010
20100130592RNA Interference Mediated Inhibition of GRB2 Associated Binding Protein (GAB2) Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating GRB2 associated binding protein (GAB2) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of GRB2 associated binding protein (GAB2) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of GAB2 genes. The small nucleic acid molecules are useful in the treatment of cancer, malignant blood disease (leukemia), inflammatory diseases or conditions, allergic diseases or conditions, or proliferative diseases or conditions.05-27-2010
20110009471Oligonucleotide analogues and methods utilizing the same - A method for the prevention or treatment in a mammal of a disease preventable or treatable by the pharmacologically useful antisense or antigene activity of an oligonucleotide analogue or a pharmacologically acceptable salt thereof in the body of said mammal, which method comprises administering to said mammal in need of such prevention or treatment a pharmaceutically effective amount of an oligonucleotide analogue comprising two or more nucleoside units, wherein at least one of said nucleoside units is a structure of the formula (2):01-13-2011
20130090367Compositions and Methods for Inhibiting Human Host Factors Required for Influenza Virus Replication - This application relates to the modulation of host cell factors required for influenza virus replication. The application relates to compounds, including nucleic acid compounds (such as, e.g., small interfering RNAs (siRNAs)) and small molecules, that target human host cell factors involved in influenza virus replication, and the use of such compounds for modulating influenza virus replication and as antiviral agents. The application also relates to methods of treating an influenza virus infection and methods of treating or preventing a symptom or disease associated with influenza virus infection, comprising administering to a subject a composition comprising a compound, such as a nucleic acid compound (e.g., an siRNA) or small molecule, that targets a human host cell factor involved in influenza virus replication.04-11-2013
20090306182RNA INTERFERENCE MEDIATED INHIBITION OF MAP KINASE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating mitogen activated protein kinase (MAP kinase) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of MAP kinase gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of MAP kinase genes, such as Jun amino-terminal kinase (e.g., JNK-1, JNK-2), p38 (MAPK 14), ERK (e.g., ERK-1, ERK-2) and/or c-Jun.12-10-2009
20090306181Compositions and methods for evaluating and treating heart failure - The invention relates to compositions, formulations, kits, and methods useful for the treatment and evaluation of heart disease in an individual.12-10-2009
20090306180COMPOUNDS AND METHODS FOR MODULATING EXPRESSION APOB - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.12-10-2009
20090306179COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF GCGR - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.12-10-2009
20090306176Use Of Low Doses Of Oligonucleotides Antisense To TGF-Beta, VEGF, Interleukin-10, C-Jun, C-Fos Or Prostaglandin E2 Genes In The Treatment Of Tumors - This invention is related to the use of at least one oligonucleotide with a length of from about 8 to about 30 nucleotide building blocks for manufacturing a pharmaceutical preparation for the prophylaxis and/or the treatment of diseases, that are modulated by TGF-beta2, TGF-beta1, TGF-beta3, VEGF, interleukin-10, c-jun, c-fos, and/or prostaglandin E2 in a mammal, wherein said oligonucleotide hybridizes with a messenger RNA of a TGF-beta2, TGF-beta1, TGF-beta3, VEGF, interleukin-10, c-jun, c-fos and/or prostaglandin E2 gene and wherein said preparation comprises said oligonucleotide in a concentration of about 1 microM to about 25 microM.12-10-2009
20090306183Compositions and Methods for the Treatment of Diseases Associated with Aberrant Cilia Assembly and Regulation - Compositions and methods are provided for identifying agents which have efficacy for the treatment of disorders related to aberrant cilial structure and function, including polycystic kidney disease.12-10-2009
20090306177Modulation of Immunostimulatory Properties of Short Interfering Ribonucleic Acid (Sirna) by Nucleotide Modification - Double-stranded short interfering ribonucleic acid (siRNA) are modified to reduce or eliminate their immunostimulatory effect without significantly affecting their gene silencing effect. Modified siRNA include one or more 2′ sugar modifications and, optionally, internucleotide linkages on the sense strand. Compositions containing the modified siRNA and methods of making and using the modified siRNA are disclosed. New and previously characterized siRNA can be synthesized to incorporate modifications according to the invention.12-10-2009
20120220647NANO-HYBRID OF TARGETABLE SIRNA-LAYERED INORGANIC HYDROXIDE, MANUFACTURING METHOD THEREOF, AND PHARMACEUTICAL COMPOSITION FOR TREATING TUMOR COMPRISING THE NANO-HYBRID - A nanohybrid of the potent gene therapeutic agent siRNA (small interfering RNA) and a target-specific layered inorganic hydroxide, a preparation method thereof, and a pharmaceutical composition for tumor treatment containing the target-specific, siRNA/layered inorganic hydroxide nanohybrid. The nanohybrid increases the in vivo stability of the siRNA, and a target-specific multifunctional ligand, which is bonded to the layered inorganic hydroxide and can bind specifically to a tumor, increases the efficiency of tumor-specific transfer of the siRNA such that the siRNA shows tumor therapeutic activity even at a relatively low dose. Thus, the nanohybrid will be widely useful for target-specific antitumor therapies.08-30-2012
20130072546TREATMENT OF METHIONINE SULFOXIDE REDUCTASE A (MSRA) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO MSRA - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Methionine Sulfoxide Reductase A (MSRA), in particular, by targeting natural antisense polynucleotides of Methionine Sulfoxide Reductase A (MSRA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of MSRA.03-21-2013
20130072544DETECTION OF HBX/8P11 HYBRID SEQUENCE IN HUMAN HEPATOCELLULAR CARCINOMA - The present invention provides a method for diagnosing a particular type of human hepatocellular carcinoma (HCC), HBx/8p11-positive HCC, in a subject by detecting the presence of a specific, non-naturally occurring polynucleotide sequence that indicates integration of a portion of the human hepatitis B virus (HBV) sequence into the human genome on chromosome 8 in the 8p11 integration region. A kit and device useful for such a method are also provided. In addition, the present invention provides a method for treating an HBx/8p11-positive HCC.03-21-2013
20130072545MiRNA MOLECULE DEFINED BY ITS SOURCE AND ITS DIAGNOSTIC AND THERAPEUTIC USES IN DISEASES OR CONDITIONS ASSOCIATED WITH EMT - The invention relates to the diagnostic and therapeutic uses of a miRNA molecule or an equivalent thereof wherein a source of said miRNA molecule or equivalent thereof comprises at least 80 nucleotides and comprises a motif having at least 98% identity with the motif represented by SEQ ID NO:1 or a source thereof in a disease and condition associated with EMT (Epithelial to Mesenchymal Transition).03-21-2013
20130072543Compounds and Compositions for Nucleic Acid Formulation and Delivery - The invention relates to compositions containing compounds of formula I:03-21-2013
20130072541ADENO-ASSOCIATED VIRAL VECTOR FOR EXON SKIPPING IN A GENE ENCODING A DISPENSIBLE-DOMAIN PROTEIN - The invention concerns an adeno-associated viral vector comprising: 03-21-2013
20130072540RNA interference of galectin-3 expression and methods of use thereof - Galectin-3 is a pro-inflammatory molecule functioning as a cytokine hub, and also regulates unfolded protein responses (UPR) and ER stress. Thus, galectin-3 serves as a target for ameliorating inflammatory diseases such as allergic inflammation and diabetic inflammation and insulin resistance. RNA interference of endogenous galectin-3 expression, upregulates IL-12, IL-10 while downregulating IL-23 production, which offers protection against allergic inflammation. In addition, endogenous galectin-3 knockdown causes upregulation of XBP1, alleviating ER stress. Together, upregulated XBP1 and IL-10 offer protection against obesity-induced inflammation. Therefore, the embodiment of the invention resides in RNA interference of endogenous galectin-3 in appropriate cell types in order to rectify allergic and/or diabetic inflammation.03-21-2013
20130072542PISCINE REOVIRUS DIAGNOSTIC COMPOSITIONS - The invention is directed to a isolated a Piscine reovirus associated with HSMI in teleosts, and isolated nucleic acids sequences and peptides thereof. The invention also relates to diagnostic antibodies against antigens derived from Piscine re-oviruses. In another aspect, the invention relates to iRNAs which target nucleic acid sequences of Piscine reoviruses. In another aspect, the invention is related to methods for detecting the presence or absence of Piscine reoviruses in an animal.03-21-2013
20110060028COMBINATION THERAPY - This disclosure relates to a SIRT1 polypeptide and a treatment regime that inhibits the activity of SIRT1 and including a method of diagnosis.03-10-2011
20130059904METHODS, AGENTS, AND COMPOUND SCREENING ASSAYS FOR INDUCING DIFFERENTIATION OF UNDIFFERENTIATED MAMMALIAN CELLS INTO OSTEOBLASTS - The present invention relates to methods, agents and compound screening assays for inducing differentiation of undifferentiated mammalian cells into osteoblasts. The invention thus provides a method, comprising contacting a compound with a polypeptide comprising an amino acid sequence selected from the group consisting of SEQ ID No: 194-309; and measuring a compound-polypeptide property related to the differentiation of said cells. The invention further relates to a bone formation enhancing pharmaceutical composition, and the use thereof in treating and/or preventing a disease involving a systemic or local decrease in mean bone density in a subject. Furthermore, the invention relates to a method for the in vitro production of bone tissue.03-07-2013
20130059903Compositions and Methods for Characterizing Breast Cancer - The invention provides compositions and methods for characterizing breast cancer stem In particular, the invention provides for the identification of cells expressing Twist and CD44 that express little or virtually undetectable levels of CD24 (i.e. a Twist03-07-2013
20130059901Pre-MRNA Trans-Splicing Molecule (RTM) Molecules and Their Uses - The present invention relates to specific and markedly improved pre-mRNA trans-splicing molecule (RTM) molecules which are designed to correct specific genes expressed within cells to be targeted, and which are associated with epidermolysis bullosa, cystic fibrosis, pachyonychia congenital, and psoriasis or neurodermitis, as well as cancers of the skin. In particular, the RTMs of the present invention are genetically engineered to interact with a specific target pre-mRNA expressed in cells to be targeted so as to result in correction of genetic defects or reprogramming of gene expression responsible for a variety of different skin disorders.03-07-2013
20110015254PTPH1 Inhibitors for the Treatment of Alzheimer's Disease - The present invention relates to the use of PTPH1 inhibitors in the prevention or treatment of Alzheimer's Disease, or a symptom thereof. The present invention also relates to a method of identifying compounds useful in the prevention or treatment of Alzheimer's Disease, or a symptom thereof.01-20-2011
20110015253ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.01-20-2011
20110015251RNA INTERFERENCE MEDIATED INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS (HIV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of human immunodeficiency virus (HIV) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HIV genes. The small nucleic acid molecules are useful in the treatment of HIV infection, AIDS, and/or disease and conditions related to HIV infection and/or AIDS in a subject or organism.01-20-2011
20110015250COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Eg5 gene (Eg5 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the Eg5 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Eg5 expression and the expression of the Eg5 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Eg5 gene in a cell.01-20-2011
20110015249METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER AND OTHER ANGIOGENESIS-RELATED DISEASES - The present invention provides nucleic acid molecules that modulate the expression of molecules in the angiopoietin/Tie2 signaling pathway. Methods of using the nucleic acid molecules are also provided.01-20-2011
20130059902METHODS AND COMPOSITIONS USEFUL IN TREATMENT OF DISEASES OR CONDITIONS RELATED TO REPEAT EXPANSION - The present invention is drawn to chemically-modified oligomers that are complementary to, and capable of hybridizing within the repeat region of CAG, CUG, or CCUG nucleotide repeat-containing RNAs (NRRs).03-07-2013
20110065774CHEMICALLY MODIFIED OLIGONUCLEOTIDES AND USES THEREOF - This invention relates generally to chemically oligonucleotides (e.g., modified oligonucleotides) useful for augmenting activity of a target gene.03-17-2011
20100099737COMPOSITIONS AND METHODS FOR TREATING MYELOSUPPRESSION - The invention provides, in part, compositions and methods for protecting a hemopoietic cell, or for treating myelosuppression, in a subject in need thereof, by administering an effective amount of an inhibitor of a SH2-containing inositol-5′-phosphatase.04-22-2010
20090298910REGULATION OF EPIGENETIC CONTROL OF GENE EXPRESSION - Methods are provided for the identification of compounds that selectively modulate epigenetic changes in gene expression. Compounds, compositions, kits or assays devices, and methods are provided for modulating the expression, endogenous levels or the function of small non-coding RNAs cognate to or transcribed by heterochromatic regions subject to epigenetic regulation (i.e., promoters, enhancers, centromeres, telomeres, origins of DNA replication, imprinted loci, or loci marked by dosage-compensation), and for modulating the formation or function of heterochromatin in cells, tissues or animals.12-03-2009
20090275633Novel Tumour Suppressor - A method of diagnosing cancer includes the steps of, in a sample obtained from a subject, determining the level or activity of HDAC2. A reduced level or activity of HDAC2 is indicative of cancer. HDAC2 protein expression is preferably determined. Indirect determination of HDAC2 expression is also possible, preferably by looking at the expression of genes selected from NCOA4, CTSB, TBCD, PPP2R4 and CORO1C. Methods for predicting the probability of successful treatment of cancer using a hydroxamic acid based HDAC inhibitor and for selecting suitable cancer treatment regimens can also be based upon determining the level or activity of HDAC2. Methods of treatment of cancer may be based upon use of carboxylic acid based HDAC inhibitors or by reconstituting HDAC2 activity.11-05-2009
20090270480Markers and Methods for Assessing and Treating Psoriasis and Related Disorders - A method for prognostic or diagnostic assessment of a skin-related disorder, such as psoriasis, in a subject correlates the presence, absence, and/or magnitude of a gene in a sample with a reference standard to determine the presence and/or severity of the disorder, and/or the response to treatment for the disorder. The method enables identification of the effectiveness of candidate therapies.10-29-2009
20090270479Genetic and Epigenetic Alterations In the Diagnosis and Treatment of Cancer - Methylation of DNA in regions involved in transcriptional regulation can induce the binding of ICBP90 and the subsequent formation of multiprotein complexes which alter gene transcription. DNA methylation in tumor suppressor genes, or in other genes which are involved in mitigating tumorigenesis, can induce binding of ICBP90 to those genes. Bound ICBP90 can interact with a pRb2/p130 regulatory complexes to remodel chromatin and inhibit transcription of the gene. DNA methyltransferases, ICBP90, and the proteins comprising the pRb2/p130 complex are therefore therapeutic targets for the treatment of cancer. Abnormalities in these proteins can also be markers of cancerous or precancerous conditions.10-29-2009
20090270481MODIFIED siRNA MOLECULES AND USES THEREOF - The present invention provides chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression. Advantageously, the modified siRNA of the present invention is less immunostimulatory than its corresponding unmodified siRNA sequence and retains RNAi activity against the target sequence. The present invention also provides nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The present invention further provides methods of silencing gene expression by administering a modified siRNA to a mammalian subject. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided.10-29-2009
20100093831NUCLEIC ACID COMPOUNDS FOR INHIBITING ANGIOGENESIS AND TUMOR GROWTH - In certain embodiments, this present disclosure provides nucleic acid compounds, compositions, and methods for inhibiting Ephrin B2 or EphB4 expression. In certain embodiments, the present disclosure provides methods and compositions for treating cancer or for treating angiogenesis-associated diseases.04-15-2010
20120225928CANCER CELL-SPECIFIC APOPTOSIS-INDUCING AGENTS THAT TARGET CHROMOSOME STABILIZATION-ASSOCIATED GENES - The present inventors discovered that inhibition of the expression of various genes associated with chromosome stabilization induces cancer cell-specific apoptosis and inhibits cell proliferation. Compounds that inhibit expression of a gene associated with chromosome stabilization or inhibit the function of a protein encoded by such a gene are thought to have cancer cell-specific apoptosis-inducing effects.09-06-2012
20120225927COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TRANSTHYRETIN - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.09-06-2012
20120225925Micro-RNA Biomarkers and Methods of Using Same - A procedure and an apparatus are described for identifying individuals at risk of pulmonary tumour and/or for diagnosing a pulmonary tumour using the study of levels of expression of miRNA in the blood or another biological fluid. Also described are a method and a compound for reducing or eliminating a risk of pulmonary tumour by rebalancing the miRNAs that are underexpressed or overexpressed.09-06-2012
20120225924SINGLE-MONOMER DERIVED LINEAR-LIKE COPOLYMER COMPRISING POLYETHYLENIMINE AND POLY(ETHYLENE GLYCOL) FOR NUCLEIC ACID DELIVERY - A method of synthesizing a random copolymer of polyethyleneimine and polyethylene glycol, comprising exposing ethanolamine in a solution to electromagnetic radiation for a sufficient time to polymerize the ethanolamine (OHCH09-06-2012
20100292298METHOD AND REAGENTS FOR TREATING HEPATIC FIBROSIS AND INFLAMMATION - The invention relates to methods for identifying an anti-fibrotic or anti-inflammatory agent comprising determining cathepsin S expression in activated hepatic stellate cells which have been exposed to a test compound and comparing expression to non-exposed hepatic stellate cells. The invention also relates to methods for treating a disorder characterised or caused by hepatic fibrosis or inflammation, comprising administering a cathepsin S inhibitor to a subject.11-18-2010
20130065947TREATMENT OF PAR4 RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO PAR4 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of PAR4, in particular, by targeting natural antisense polynucleotides of PAR4. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of PAR4.03-14-2013
20130065946Materials and Methods Related to Modulation of Mismatch Repair and Genomic Stability by miR-155 - The present invention provides materials and methods related to modulation of mismatch and genomic stability by miR-155.03-14-2013
20130065945NLK AS A MARKER FOR DIAGNOSIS OF LIVER CANCER AND AS A THERAPEUTIC AGENT THEREOF - A novel marker for diagnosis of liver cancer and use thereof are provided. To be specific, a marker for diagnosis of liver cancer using over-expression of NLK (neuro-like kinase) in liver cancer cell is provided, along with a composition for diagnosis of liver cancer, a kit, a microarray, and a method for diagnosing liver cancer using the marker. Additionally, a method for screening a substance to prevent or treat liver cancer by decreasing expression of the marker gene or protein, and a composition for preventing or treating liver cancer including such substance are provided. Accordingly, the NLK gene can be efficiently used as a target for diagnosis and treatment of liver cancer.03-14-2013
20130065944REVERSE MICELLE SYSTEM COMPRISING NUCLEIC ACIDS AND USE THEREOF - The present invention relates to reverse micelle system based on sterols, acylglycerols, phospholipids or sphingolipids and nucleic acids. The reverse micelle system of the invention is able to cross mucosa and cellular membranes. It thus allows vectorization of nucleic acids to target sites. It is advantageously useful in the pharmaceutical and dietetic fields.03-14-2013
20130065943COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF CD45 GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the CD45 gene.03-14-2013
20130065942Nucleic Acid-Lipopolymer Compositions - Compositions, methods, and applications that increase the efficiency of nucleic acid transfection are provided. In one aspect, a pharmaceutical composition may include at least about 0.5 mg/ml concentration of a nucleic acid condensed with a cationic lipopolymer suspended in an isotonic solution, where the cationic lipopolymer includes a cationic polymer backbone having cholesterol and polyethylene glycol covalently attached thereto, and wherein the molar ratio of cholesterol to cationic polymer backbone is within a range of from about 0.1 to about 10, and the molar ratio of polyethylene glycol to cationic polymer backbone is within a range of from about 0.1 to about 10. The composition further may include a filler excipient.03-14-2013
20130065941COMPOSITIONS AND THEIR USES FOR GENE THERAPY OF BONE CONDITIONS - In certain preferred embodiments, the present invention provides compositions and methods for the treatment of bone conditions associated with low bone density. In preferred embodiments, the present invention provides compositions and methods for the treatment of osteoprotegerin-responsive conditions.03-14-2013
20130065940DOUBLE-STRANDED RNA MOLECULES WITH STABILITY IN MAMMALIAN BODY FLUID, PREPARATION AND APPLICATION THEREOF - The present invention discloses preparation and application of double-stranded RNA molecules stable in mammalian body fluids. The mammalian-body-fluid-stable RNA molecules disclosed in the present invention are comprised of only unmodifide nucleotides. For the first time, the present invention discloses the applications of mammalian-body-fluid-stable RNA molecules for immunotherapy and siRNA drug development.03-14-2013
20130065938Mutator Activity Induced by microRNA-155 (miR-155) Links Inflammation and Cancer - Methods of reducing spontaneous mutation rate of a cell in a subject in need thereof by reducing endogenous levels of miR-155 are described.03-14-2013
20130065939COMPOSITIONS AND METHODS FOR SILENCING GENES EXPRESSED IN CANCER - The present invention provides therapeutic nucleic acids such as interfering RNA (e.g., siRNA) that target the expression of genes associated with tumorigenesis and/or cell transformation, lipid particles (e.g., nucleic acid-lipid particles) comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles, e.g., for the treatment of a cell proliferative disorder such as cancer.03-14-2013
20110021610AROMATIC PRENYLTRANSFERASE FROM HOP - Nucleic acid molecules from hop (01-27-2011
20110021609MicroRNA Signatures Associated with Cytogenetics and Prognosis in Acute Myeloid Leukemia (AML) and Uses Thereof - Methods and compositions utilizing an miRNA signature for the diagnosis, prognosis and/or treatment of leukemia associated diseases, particularly acute myeloid leukemia, are disclosed.01-27-2011
20110021607Methods and Compositions Relating to Carcinoma Stem Cells - MicroRNA markers of breast cancer stem cells (BCSC) are provided herein. The markers are polynucleotides that are differentially expressed in BCSC as compared to normal counterpart cells. Uses of the markers include use as targets for therapeutic intervention; as targets for drug development, and for diagnostic or prognostic methods relating to breast cancer and BCSC cell populations. BCSCs have the phenotype of having lower expression of certain miRNAs compared to normal breast epithelial cells, or to cancer cells that are not cancer stem cells.01-27-2011
20110021606RNAi Modulation of RSV, PIV and Other Respiratory Viruses and Uses Thereof - The present invention is based on the in vivo demonstration that RSV and PIV can be inhibited through intranasal administration of RNAi agents as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the present invention provides general and specific compositions and methods that are useful in reducing RSV or PIV mRNA levels, RSV or PIV protein levels and viral titers in a subject, e.g., a mammal, such as a human. These findings can be applied to other respiratory viruses.01-27-2011
20110021605MEANS AND METHODS FOR THE SPECIFIC INHIBITION OF GENES IN CELLS AND TISSUE OF THE CNS AND/OR EYE - Described is a method for the specific modulation of the expression of target genes in cells and/or tissues of the CNS and/or eye, wherein a composition comprising one or more doubled stranded oligoribonucleotides (dsRNA) is introduced into the cell, tissue or organism outside the blood-brain or blood-retina barriers. Furthermore, a method for the identification and validation of the function of a gene is provided, wherein the method provides a test cell, test tissue or test organism, which allow information to be gained on the function of the target gene. In addition, compositions and kits are described useful for those methods. In particular, components and methods for the diagnostic use and/or therapy of disorders related to the CNS and/or eye are provided which are based on RNA interference.01-27-2011
20110021604METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF KRAS BY ASYMMETRIC DOUBLE-STRANDED RNA - This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer substrate siRNA (DsiRNA) agents possessing asymmetric end structures.01-27-2011
20110021603TRPM-2 Antisense Therapy - It has now been determined that antisense therapy which reduces the expression of TRPM-2 provides therapeutic benefits in the treatment of cancer. In particular, such antisense therapy can be applied in treatment of prostate cancer and renal cell cancer. Addition of antisense TRPM-2 ODN to prostatic tumor cells in vivo is effective for delaying the onset of androgen independence. Thus, prostate cancer can be treated in an individual suffering from prostate cancer by initiating androgen-withdrawal to induce apoptotic cell death of prostatic tumor cells in the individual, and administering to the individual a composition effective to inhibit expression of TRPM-2 by the tumor cells, thereby delaying the progression of prostatic tumor cells to an androgen-independent state in an individual Combined use of antisense TRPM-2 and taxanes synergistically enhances cytotoxic chemosensitivity of androgen-independent prostate cancer. In addition, it has also been found that antisense TRPM-2 has beneficial effect for other cancer types. Specifically, antisense TRPM-2 ODN enhances chemosensitivity in human Renal cell cancer, a normally chemoresistant disease with no active chemotherapeutic agent having an objective response rate higher than 10%. Radiation sensitivity is also enhanced when cells expressing TRPM-2 are treated with antisense TRPM-2 ODN. Thus, the antisense TRPM-2 ODNs can be used to enhance hormone sensitivity, chemosensitivity and radiation sensitivity of a variety of cancer types in which expression of TRPM-2 has been observed.01-27-2011
20110021600NOVEL NUCLEIC ACID - According to the present invention, it possible to provide a novel nucleic acid, a vector that expresses the nucleic acid, a screening method for a substance that controls the nucleic acid, a method of detecting the expression and a mutation of the nucleic acid, and a diagnostic method or therapeutic method for a disease caused by an abnormality of mesenchymal stem cells, mast cells or the like, or a disease caused by a cell proliferation abnormality, such as cancer.01-27-2011
20110021599METHODS AND COMPOSITIONS FOR INHIBITING OR REDUCING HAIR LOSS, ACNE, ROSACEA, PROSTATE CANCER, AND BPH - This invention provides methods of treating androgenetic alopecia (AGA), acne, rosacea, prostate cancer, and benign prostatic hypertrophy (BPH), comprising the step of contacting a subject with a compound or composition capable of decreasing prostaglandin D2 (PGD2) level or activity, a downstream signaling or receptor pathway thereof, or prostaglandin D2 synthase level or activity; methods of stimulating hair growth, comprising the step of contacting a subject with a compound or composition capable of increasing or decreasing the activity or level of a target gene of the present invention, or with a protein product of the target gene or an analogue or mimetic thereof; and methods of testing for AGA and evaluating therapeutic methods thereof, comprising measuring PGD2 levels.01-27-2011
20090253774RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET DERIVED GROWTH FACTOR (PDGF) AND PLATELET DERIVED GROWTH FACTOR RECEPTOR (PDGFR) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating platelet derived growth factor (PDGF) and/or platelet derived growth factor receptor (PDGFr) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of platelet derived growth factor (PDGF) and/or platelet derived growth factor receptor (PDGFr) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of platelet derived growth factor (PDGF) and/or platelet derived growth factor receptor (PDGFr) genes, such as PDGF and/or PDGFr.10-08-2009
20120115935ANTI-CANCER AGENT, METHOD FOR INDUCING APOPTOSIS OF CANCER CELLS, AND METHOD FOR SCREENING FOR ANTI-CANCER AGENT - Disclosed is an anti-cancer agent which can inhibit the progression of cell cycle in the M phase, unlike taxane anti-cancer agents. The anti-cancer agent comprises a compound capable of inhibiting specifically the expression and/or functions of RBM8A depicted in SEQ ID NO: 1, Magoh depicted in SEQ ID NO: 3 or an isoform thereof. The anti-cancer agent can inhibit the expression and/or functions of RBM8A or Magoh in cancer cells to terminate the cell cycle in the M phase, thereby inhibiting the proliferation of the cells. Therefore, the anti-cancer agent can inhibit the proliferation of cancer cells and induce the apoptosis of cancer cells, leading to the cure of cancer.05-10-2012
20120115932MODULATION OF STAT 6 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of STAT 6. The compositions comprise oligonucleotides, targeted to nucleic acid encoding STAT 6. Methods of using these compounds for modulation of STAT 6 expression and for diagnosis and treatment of disease associated with expression of STAT 6 are provided.05-10-2012
20120115931SELECTIVE INHIBITORS OF CB2 RECEPTOR EXPRESSION AND/OR ACTIVITY FOR THE TREATMENT OF OBESITY AND OBESITY-RELATED DISORDERS - The invention relates to a method for treating and/or preventing obesity and/or obesity-related disorders by administering to a subject in need thereof a selective inhibitor of cannabinoid type 2 (CB2) receptor expression.05-10-2012
20120115930COMPOSITIONS AND THEIR USES DIRECTED TO HEPCIDIN - Disclosed herein are compounds, compositions and methods for modulating the expression of hepcidin in a cell, tissue or animal or preventing, ameliorating or treating anemia. Also provided are methods for prevention, amelioration or treatment of anemia, and for increasing red blood cell count in an animal. Also provided are methods for the prevention, amelioration and/or treatment of low serum iron levels, low red blood cell count and other clinical endpoints of anemia in an animal. These methods may be achieved by administration of compounds or compositions including antisense compounds targeted to a nucleic acid that expresses hepcidin polypeptide combined with an erythropoiesis stimulating agent.05-10-2012
20120115928MIRNA AND ITS TARGETS RESPECTIVELY THE PROTEINS MADE BASED ON THE TARGETS AS A PROGNOSTIC, DIAGNOSTIC BIOMARKER AND THERAPEUTIC AGENT FOR CANCER - A compound miRNA (miRNA661) (Nr MI0003669 or ENSG00000207574) the sequence of the mature miRNA-661 being 51-ugccugggucucuggccugcgcgu-74 for use as a medicament in the in the treatment and/or the diagnosis of cancer, neuronal disease and infection.05-10-2012
20120115926ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION - Methods for the rapid and long-term lowering of lipid levels in human subjects and for the treatment of conditions associated with elevated LDL-cholesterol and elevated apolipoprotein B are provided.05-10-2012
20120115924Micro-RNA Mediated Modulation of Colony Stimulating Factors - The present invention relates to the modulation of immunoregulatory proteins, including cytokines, such as colony stimulatory factors (CSF) via the use of microRNA-155 modulators.05-10-2012
20120115923Sirna Compositions Promoting Scar-Free Wound Healing of Skin and Methods for Wound Treatment - This invention describes compositions and methods using siRNA to target various genes expressed in cells of injured tissue during scar formation to promote scar-free wound healing.05-10-2012
20090215862Micro rna - Micro RNA capable of interacting with the 3′ untranslated region of kit protein mRNA is useful in treating kit-dependent tumours, and inhibitors therefor are useful in treating suppressed haematopoiesis in cancer patients or abnormal erythropoiesis in β-thalassemia, for example.08-27-2009
20090239935RNA-HELICASE AS A MARKER FOR RARE TUMORS - Method for the diagnosis of diseases in a mammal, preferably in a human, wherein a probe from the mammal is examined with a view to an elevated level of RNA helicase and the elevated level of RNA helicase indicates the disease. The disease refers to esophagus carcinoma, pancreas carcinoma, stomach carcinoma, hepatocellular carcinoma, hepatoblastoma and cholangiocellular carcinoma.09-24-2009
20090306185Nanogenomics for medicine: siRNA engineering - Described herein are materials and methods for the delivery of siRNA and the production of nanoparticles useful for the delivery of siRNA. Methods of treating a disease or disorder using the nanoparticles described herein also are disclosed.12-10-2009
20120232127REGULATION OF NEUROTRANSMITTER RELEASE THROUGH ANION CHANNELS - A novel use of anion channels, preferably Ca09-13-2012
20100087507USE OF RPN2 GENE EXPRESSION INHIBITOR - The present invention uses an RPN2 gene expression inhibitor as a cancer cell growth inhibitor, which further includes a drug showing an anti-cancer action if desired, and is administered in combination with atelocollagen if desired. In addition, the present invention is an anti-cancer agent including such cancer cell growth inhibitor.04-08-2010
20100087509Methods, Agents, and Compound Screening Assays for Inducing Differentiation of Undifferentiated Mammalian Cells into Osteoblasts - The present invention relates to methods, agents and compound screening assays for inducing differentiation of undifferentiated mammalian cells into osteoblasts. The invention thus provides a method, comprising contacting a compound with a polypeptide comprising an amino acid sequence selected from the group consisting of SEQ ID No: 194-309; and measuring a compound-polypeptide property related to the differentiation of said cells. The invention further relates to a bone formation enhancing pharmaceutical composition, and the use thereof in treating and/or preventing a disease involving a systemic or local decrease in mean bone density in a subject. Furthermore, the invention relates to a method for the in vitro production of bone tissue.04-08-2010
20120238619MICRO-RNA FOR THE REGULATION OF CARDIAC APOPTOSIS AND CONTRACTILE FUNCTION - The present invention relates to treating or preventing age-related cardiomyopathy by modulating the expression or activity of a miR-34 family member and/or PNUTS. Methods of treating or preventing age-related cardiomyopathy include administering an inhibitor of miR-34 expression or activity or an agonist of PNUTS expression or activity. Also provided herein are methods of treating or preventing cardiac fibrosis and myocardial infarction by administering an inhibitor of miR-34 expression or activity or an agonist of PNUTS expression or activity.09-20-2012
20120238618Pharmaceutical Composition Comprising Anti-miRNA Antisense Oligonucleotides - The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 26 nucleobases which are complementary to human microRNAs selected from the group consisting of miR19b, miR21, miR122a, miR155 and miR375. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogues into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC.09-20-2012
20120238617MICRORNA EXPRESSION SIGNATURE IN PERIPHERAL BLOOD OF PATIENTS AFFECTED BY HEPATOCARCINOMA OR HEPATIC CIRRHOSIS AND USES THEREOF - A method for diagnosing or prognosticating hepatocellular carcinoma, also in the early stages, or for assessing the risk of developing hepatocellular carcinoma, or for monitoring the effectiveness of an anti-tumour therapy against hepatocellular carcinoma by measuring the expression level of at least one miRNA gene product in a peripheral blood sample or in a biological fluid sample. Said method comprises measuring, in an isolated sample of peripheral blood or biological fluid, the expression level of at least one miRNA gene product, and comparing said measured expression level with a reference level. Such method can also be used to diagnose or assess the risk of developing liver cirrhosis in patients affected by chronic hepatitis, or to prognosticate the evolution of cirrhosis in patients affected by cirrhosis, or to monitor the effectiveness of a pharmacological therapy against liver cirrhosis.09-20-2012
20130165496COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF GCCR - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.06-27-2013
20130165497MONITORING OF IMMUNE SYSTEM USING PERIPHERAL BLOOD MICRO-RNA EXPRESSION PROFILE ANALYSIS AND USES THEREOF - The present invention relates to a method for monitoring the immune system of an individual, which comprises measuring, preferably by quantitative RT-PCR, the expression level of at least one microRNA (miRNA) gene product in a peripheral blood sample or in a biological fluid sample, and comparing said measured expression level with a reference level. In particular, the at least one miRNA gene product, which the method of the invention measures, is expressed by lymphocyte populations of an individual, in particular by naive CD4+T, TH1, TH2 and TH17 lymphocytes. The method of the invention is useful for the diagnosis, prognosis, prevention, control and/or the treatment of a pathological condition caused by or associated with an immune system dysfunction. Moreover, the method of the present invention is useful for monitoring, in an individual, the evolution of conditions mediated by the immune system, such as the response to a vaccination.06-27-2013
20130165498INVERTEBRATE MICRORNAS - This invention provides plants having resistance to invertebrate pests. More specifically, this invention discloses a non-natural transgenic plant cell expressing at least one invertebrate miRNA in planta for suppression of a target gene of an invertebrate pest or of a symbiont associated with the invertebrate pest. Also provided are recombinant DNA constructs for expression of at least one invertebrate miRNA in planta, a non-natural transgenic plant containing the non-natural transgenic plant cell of this invention, a non-natural transgenic plant grown from the non-natural transgenic plant cell of this invention, and non-natural transgenic seed produced by the non-natural transgenic plants, as well as commodity products produced from a non-natural transgenic plant cell, plant, or seed of this invention. This invention further provides a method of suppressing at least one target gene of an invertebrate pest of a plant or of a symbiont associated with the invertebrate, including providing a plant including the non-natural transgenic plant cell of this invention, wherein the invertebrate is the invertebrate pest, the recombinant DNA is transcribed in the non-natural transgenic plant cell to the recombinant miRNA precursor, and when the invertebrate pest ingests the recombinant miRNA precursor, the at least one target gene is suppressed.06-27-2013
20130165499Methods And Compositions For Prevention Or Treatment Of RSV Infection - Methods and compositions are provided for the prevention or treatment of RSV infection in a human. The methods include administering one or more doses of a composition comprising an siRNA. The dose can be formulated for topical or parenteral administration. Topical administration includes administration as a nasal spray, or by inhalation of respirable particles or droplets.06-27-2013
20130165500RNA Interference Mediated Inhibition of Prolyl Hydroxylase Domain 2 (PHD2) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond expressed/synthetic to the modulation of PHD2 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short inter-fering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsR-NA), micro-RNA (miRNA), and short hair-pin RNA (shRNA) molecules that are capa-ble of mediating or that mediate RNA inter-ference (RNAi) against PHD2 gene expres-sion.06-27-2013
20120095075NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS - The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure:04-19-2012
20110152355METHOD FOR PREDICTING AND DETECTING TUMOR METASTASIS - The invention provides a method of determining the prognosis of cancer in a subject. The method comprises (a) obtaining a sample from the subject, (b) analyzing the sample for the expression level of a carboxypeptidase E (CPE) splice variant, and (c) correlating the expression level in the sample with the prognosis of cancer in the subject. The invention further provides a method of diagnosing cancer, methods of treatment, kits for detecting mRNA expression of a CPE-ΔN, and inhibitors of CPE-ΔN and compositions thereof.06-23-2011
20100286233PERIPHERAL AND NEURAL INFLAMMATORY CROSSTALK - Disclosed are compositions and methods for the study and treatment of inflammatory disease, neurological disorders, bone disease, pain, and methods of making and using thereof.11-11-2010
20100261776BRUTON'S TYROSINE KINASE AS ANTI-CANCER DRUG TARGET - Receptor protein tyrosine kinases (RPTKs) transmit extracellular signals across the plasma membrane to cytosolic proteins, stimulating the formation of complexes that regulate key cellular functions. Over half of the 90 tyrosine kinases have been implicated in human cancers and are for this reason considered highly promising drug targets. To gain insight into the tyrosine kinases that contribute to breast cancer related cellular mechanisms, we carried out a large-scale loss-of-function analysis of the tyrosine kinases, using RNA interference, in the clinically relevant Erb-B2 positive, BT474 breast cancer cell line. The cytosolic, non-receptor tyrosine kinase Bruton's tyrosine kinase (BTK), which has been extensively studied for its role in B cell development, was among those tyrosine kinase genes required for BT474 breast cancer cell survival. The BTK protein identified was an alternative form containing an amino-terminal extension. This alternative form of the Btk message is also present in tumorigenic breast cells at significantly higher levels than in normal breast cells.10-14-2010
20100324112Genetic changes in ATM and ATR/CHEK1 as prognostic indicators in cancer - The present invention relates to the discovery that, in human cancer, an 11q deletion of ATM together with an increase in ATR and CHEK1 expression correlates with resistance to ionizing radiation which could be overcome by inhibition of the ATR/CHEK1 pathway. It provides for methods of identifying patients unlikely to exhibit an adequate response to radiation therapy and/or chemotherapy who may benefit from ATR/CHEK1 pathway inhibition, as well as methods of treating said patients.12-23-2010
20120077865METHODS FOR TREATING HYPERCHOLESTEROLEMIA - Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.03-29-2012
20120136046METHOD OF TREATING AGE RELATED DISORDERS - The present invention relates to the use of fujimycin for the treatment of a disorder related to the chronological and/or replicative life-span of a cell, and to methods for increasing the replicative life span of a cell, said method comprising disrupting the function of a polynucleotide or gene encoding a polypeptide comprising SEQ ID No: 1, 3 or 5.05-31-2012
20100286228Method of inhibiting intimal hyperplasia - The present invention relates, in general, to intimal hyperplasia, and, in particular, to a method of inhibiting intimal hyperplasia using siRNA to E2F. The invention further relates to compounds and compositions suitable for use in such a method.11-11-2010
20120329857Short Hairpin RNAs for Inhibition of Gene Expression - Methods, compositions, and kits that include small hairpin RNA (shRNA) useful for inhibition of gene expression, such as viral-mediated gene expression, are described.12-27-2012
20110294871LIPIDS, LIPID COMPLEXES AND USE THEREOF - The present invention is related to a compound according to formula (I),12-01-2011
20110294869SELF DELIVERING BIO-LABILE PHOSPHATE PROTECTED PRO-OLIGOS FOR OLIGONUCLEOTIDE BASED THERAPEUTICS AND MEDIATING RNA INTERFERENCE - Disclosed herein are compositions and methods for generating ribo-nucleic neutral (RNN) or deoxyribo-nucleic-neutral (DNN) polynucleotides with reduced anionic charge, for improved intracellular delivery. Also disclosed herein are methods of using RNN and DNN compositions.12-01-2011
20110294868MODULATION OF TRANSTHYRETIN EXPRESSION - Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.12-01-2011
20110294867SENSIZITATION OF CANCER CELLS TO THERAPY USING SINA TARGETING GENES FROM THE 1P AND 19Q CHROMOSOMAL REGIONS - The invention relates to the identification of genes involved in resistance of cancer cells to therapy, to short nucleic acid molecules which inhibit the expression of these genes by RNA interference and to their use as adjuvant in cancer therapy, to sensitize cancer cells to conventional anticancer agents; the short nucleic acid molecules are double-stranded short interfering nucleic acid molecules including a sense and an antisense region, wherein the sense region includes a nucleotide sequence that is selected from the group consisting of: the sequences SEQ ID NO: 15, 11, 13, 14, 30, 31, 38, 46, 64 and 70 and the sequences having at least 70% identity, preferably at least 80% identity, more preferably at least 90% identity with the sequences, and the antisense region includes a nucleotide sequence that is complementary to the sense region.12-01-2011
20110294866PHARMACEUTICAL COMPOSITION FOR TREATING DEMENTIA COMPRISING SHRNA INHIBITING S100A9 EXPRESSION - Disclosed is a composition for treating dementia including shRNA to inhibit expression of S100a9. More particularly, the present disclosure describes a composition for prevention or treatment of dementia which includes shRNA having a nucleotide sequence defined by SEQ. ID No. 1 or 2 or a mixture thereof wherein the nucleotide sequence is complementarily bonded to mRNA of S100a9 in order to inhibit expression of S100a9, as well as a method for prevention or treatment of dementia, including administering the foregoing shRNA into a mammalian cell including a human cell or in vitro established mammalian cell-line, in order to inhibit expression of S100a9 protein.12-01-2011
20100120893Compositions and Methods for Inhibiting Expression of Transthyretin - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.05-13-2010
20110046201METHODS AND COMPOSITIONS FOR SEAMLESS CLONING OF NUCLEIC ACID MOLECULES - The present invention is in the fields of biotechnology and molecular biology. More particularly, the present invention relates to cloning or subcloning one or more nucleic acid molecules comprising one or more type IIs restriction enzyme recognition sites. The present invention also embodies cloning such nucleic acid molecules using recombinational cloning methods such as those employing recombination sites and recombination proteins. The present invention also relates to nucleic acid molecules (including RNA and iRNA), as well as proteins, expressed from host cells produced using the methods of the present invention.02-24-2011
20110281931RNA SEQUENCE-SPECIFIC MEDIATORS OF RNA INTERFERENCE - The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.11-17-2011
20110009469COMPOSITIONS AND METHODS OF TREATING NEOPLASIA - The present invention provides compositions and methods featuring microRNA polynucleotides for the diagnosis, treatment or prevention of neoplasia.01-13-2011
20110144183OLIGONUCLEOTIDES FOR TREATING INFLAMMATION AND NEOPLASTIC CELL PROLIFERATION - There is provided oligonucleotides directed against the CCR3 receptor and the common beta sub-unit of IL-3, IL-5 and GM-CSF receptors. The oligonucleotides are useful to inhibit general inflammation, including inflammation associated with asthma, COPD, allergy, Cystic fibrosis (CF), hypereosinophilia and neoplastic cell proliferation such as cancer.06-16-2011
20110144182TREATMENT OF SURGICAL ADHESIONS - Connexin modulation for the treatment of surgical adhesions, and associated methods, compositions, and articles.06-16-2011
20100286244RNAi MEDIATED KNOCKDOWN OF NUMA FOR CANCER THERAPY - This invention relates to the use of short interfering nucleic acid molecules (siRNA) to inhibit Nuclear Mitotic Apparatus Protein (NuMA) gene expression and their use in treatment of disease, including cancer.11-11-2010
20110009468MIRNA, SIRNA AND USE THEREOF IN THERAPY - The present invention relates to a mixture of molecules comprising at least one miRNA and at least one siRNA, or at least two miRNAs, or at least two siRNAs for inducing hematopoietic differentiation or for treating leukemia wherein the miRNA is able to modulate hematopoietic differentiation and/or to act as oncosuppressor, and the siRNA is able to modulate hematopoietic differentiation or to inhibit the expression of a fusion product deriving from a chromosomic translocation associated to leukemia.01-13-2011
20090209618Pyruvate dehydrogenase kinases as therapeutic targets for cancer and ischemic diseases - The invention provides therapeutic and prophylactic compounds and methods for altering the activity of pyruvate dehydrogenase kinase (e.g. PDK1, PDK2, PDK3, PDK4). Such therapies are useful for the treatment of neoplasia. The invention further provides therapeutic and prophylactic compounds and methods of altering pyruvate dehydrogenase activity to treat or prevent cell death related to ischemia.08-20-2009
20100267808Bispecific Antisense Oligonucleotides that Inhibit IGFBP-2 and IGFBP-5 and Methods of Using Same - Bispecific antisense oligonucleotides which consist essentially of a sequence of bases that is complementary to portions of both the gene encoding human IGFBP-2 and the gene encoding human IGFBP-5 are useful in as antisense therapeutics in the treatment of endocrine-regulated cancers.10-21-2010
20100267807ALLEVIATING NEUROPATHIC PAIN WITH EETS AND SEH INHIBITORS - The invention discloses methods of using cis-epoxyeicosantrienoic acids (“EETs”), inhibitors of soluble epoxide hydrolase (“sEH”), or a combination of an EET and an inhibitor of sEH, to alleviate neuropathic pain in subjects suffering from such pain.10-21-2010
20100267810METHODS AND COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE - This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases.10-21-2010
20100267801CYCLOHEXYL SULPHONES FOR TREATMENT OF CANCER - Sulphones of formula (I) are disclosed for use in treatment of cancer.10-21-2010
20100222411VHZ FOR DIAGNOSIS AND TREATMENT OF CANCER - We provide VHZ for use in a method of treatment, prophylaxis or alleviation of a cancer, such as breast cancer, in an individual. We provide an anti-VHZ agent for the treatment, prophylaxis or alleviation of cancer. We further provide a kit for detecting breast cancer in an individual or susceptibility of the individual to breast cancer comprising means for detection of VHZ expression in the individual or a sample taken from him or her as well as a method of detecting a cancer cell, the method comprising detecting modulation of expression, amount or activity of VHZ in the cell.09-02-2010
20090326043Method and Compound for Antiviral (HIV) Therapy - Disclosed is an anti-viral therapeutic that inactivates the Human Immunodeficiency Virus (HIV) RNA by siDNA. The siDNA antiviral therapeutic is effective in the treatment and inactivation of cell free virus particles before infection and/or in the treatment and prevention of HIV infections inside the cell. The invention exploits the HIV RNase H activity of the reverse transcriptase which is essential for viral replication, causing premature cleavage and degradation of the viral RNA genome.12-31-2009
20110130443Compositions And Methods For Inhibiting Expression Of Factor V Leiden Mutant Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor V Leiden mutant gene (Factor V Leiden mutant gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Factor V Leiden mutant gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Factor V Leiden mutant gene using the pharmaceutical composition; and methods for inhibiting the expression of the Factor V Leiden mutant gene in a cell.06-02-2011
20110130441OLIGOMERIC COMPOUNDS HAVING AT LEAST ONE NEUTRALLY LINKED TERMINAL BICYCLIC NUCLEOSIDES - The present disclosure describes oligomeric compounds having at least one bicyclic nucleoside attached to the 3′ or 5′ termini by a neutral internucleoside linkage and methods of using the oligomeric compounds. In some embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.06-02-2011
20110136891Nucleic acid molecules and collections thereof, their application and Identification - Provided is a method for characterising a sample comprising nucleic acid derived from a cell. The method comprises determining whether a sample comprises at least a minimal sequence of at least one new microRNA (miRNA) as disclosed herein or a mammalian ortholog thereof and characterizing the sample on the basis of the presence or absence of the miRNA. The invention further provides new nucleic acid molecules and collections thereof and their use in therapeutic and diagnostic applications. The invention furthermore provides a method for identifying a miRNA molecule or a precursor molecule thereof.06-09-2011
20110136890TREATMENT OF FIBROTIC CONDITIONS - Treatment of fibrosis and fibrotic diseases, disorders, and conditions, and associated methods, compositions, formulations and articles.06-09-2011
20090209619Depression of herg k+ channel function in mammallan cells and applications to the control of cancer cells division - The use of an HERG channel inhibitor for controlling the proliferation of cancer cells. Examples of such HERG channel inhibitors include dofetilide, cisapride, E-4031 and a siRNA molecule targeting a sequence involved in the expression of an HERG channel. Other ERG channels are also targets for these inhibitors.08-20-2009
20100144835INHIBITION OF GPR4 - The present invention relates to the use of a GPR4 inhibitor for the manufacture of a medicament for the inhibition of angiogenesis, for instance for the inhibition of tumour growth in the treatment of cancer. In a preferred embodiment, said inhibitor is a siRNA, preferably double-stranded.06-10-2010
20100331390EFFECTS OF APOLIPOPROTEIN B INHIBITION ON GENE EXPRESSION PROFILES IN ANIMALS - Methods are provided for modulating the expression of genes involved in lipid metabolism, useful in the treatment of conditions associated with cardiovascular risk. Antisense oligonucleotides targeted to apolipoprotein B reduce the level of apolipoprotein B mRNA, lower serum cholesterol and shift liver gene expression profiles from those of an obese animal towards those of a lean animal. Further provided are methods for improving the cardiovascular risk of a subject through antisense inhibition of apolipoprotein B. Also provided are methods for employing antisense oligonucleotides targeted to apolipoprotein B to modulate a cellular pathway or metabolic process.12-30-2010
20100286237PRO-ANGIOGENIC GENES IN OVARIAN TUMOR ENDOTHELIAL CELL ISOLATES - A gene profiling signature for ovarian tumor endothelial cells is disclosed herein. The gene signature can be used to diagnosis or prognosis an ovarian tumor, identify agents to treat an ovarian tumor, to predict the metastatic potential of an ovarian tumor and to determine the effectiveness of ovarian tumor treatments. Thus, methods are provided for identifying agents that can be used to treat ovarian cancer, for determining the effectiveness of an ovarian tumor treatment, or to diagnose or prognose an ovarian tumor. Methods of treatment are also disclosed which include administering a composition that includes a specific binding agent that specifically binds to one of the disclosed ovarian endothelial cell tumor-associated molecules and inhibits ovarian tumor in the subject.11-11-2010
20120289583TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3) - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Insulin Receptor Substrate 2 (IRS2) polynucleotides, in particular, by targeting natural antisense polynucleotides of Insulin Receptor Substrate 2 (IRS2) polynucleotides and Transcription factor E3 (TFE3). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IRS2.11-15-2012
20110124706SOCS3 Inhibition Promotes CNS Neuron Regeneration - Regeneration of injured CNS neurons in a mammal in need thereof is promoted by delivering directly to the body of the injured CNS neurons, such as with an intracerebral or intraretinal cannula, an effective amount of a specific inhibitor of SOCS3.05-26-2011
20100292302INDUCTION OF APOPTOSIS AND INHIBITION OF CELL PROLIFERATION THROUGH MODULATION OF CARNITINE PALMITOYLTRANSFERASE 1C ACTIVITY - This invention relates to compositions and methods for cancer therapeutics. In particular, the present invention provides compositions and methods for treating tumors by inhibiting the activity of CPT1C. The methods and compositions can additionally include inhibition of glycolysis.11-18-2010
20110301218MODIFIED snRNAs FOR USE IN THERAPY - The invention relates to a nucleic acid encoding a small nuclear RNA (snRNA), which snRNA is modified so that it contains sequence capable of hybridizing with the 5′ and 3′ splice site junctions and an exonic splicing enhancer of an exon of a pre-mRNA, so that the exon sequence is skipped during the splicing process that converts the pre-mRNA in the mature mRNA. The nucleic acid, the modified snRNA and vectors incorporating the nucleic acid may be used in the therapy, in particular in the treatment of muscular dystrophies.12-08-2011
20110301224METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene.12-08-2011
20110301220RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.12-08-2011
20110301223COMPOSITIONS AND METHODS FOR INSECTICIDAL CONTROL OF STINKBUGS - Methods and compositions are provided which employ a silencing element that, when ingested by a pest, such as a Pentatomidae plant pest or a 12-08-2011
20110301222COMBINATION THERAPY FOR THE TREATMENT OF CANCER - The present invention provides methods of sensitizing cancer cells to docetaxel and inhibiting the growth of various tumors by employing a modified eIF-4E antisense oligonucleotide and docetaxel in combination.12-08-2011
20110301221DIAGNOSIS, PROGNOSIS AND TREATMENT OF GLIOBLASTOMA MULTIFORME - The present invention in one aspect relates generally to the identification, provision and use of a plurality of biomarkers to provide risk assessment of a subject having glioblastoma multiforme, and products and processes related thereto. In one aspect, a novel plurality of biomarkers as described herein is provided to determine a risk of glioblastoma multiforme. In another aspect, a novel plurality of biomarkers as described herein is provided to diagnose a subject having glioblastoma multiforme. In yet another aspect are methods for treating a subject having glioblastoma multiforme by administering one or more therapeutic regimens for glioblastoma multiforme. In yet another aspect are nucleic acid arrays comprising nucleic acid probes that hybridize to one or more glioblastoma multiforme genes.12-08-2011
20110166203ANTI-HEPATITIS C VIRUS COMPOSITION - The present invention provides an anti-hepatitis C virus composition that includes a substance that suppresses the expression or function of a PA28γ gene, a method for preventing hepatitis C viral infection or suppressing hepatitis C virus growth that includes the step of administering the composition to a subject, and a method for screening an effective component of an anti-hepatitis C virus composition that includes the step of selecting a substance that inhibits the expression or function of a PA28γ gene.07-07-2011
20110288150Oligonucleotides for Suppressing Cancer Cell Invasion and Migration - Provided herein is a method for detection of migratory and invasive cancer cells based on a number of marker nucleic acids differentially expressed in migratory/invasive cancer cells relative to nonmigratory/noninvasive cancer cells. Also disclosed are antisense oligonucleotides of the marker nucleic acids and uses thereof for suppressing cancer cell migration and invasion.11-24-2011
20110288147Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop - The invention features compositions and methods that are useful for reducing the expression or activity of a specified gene in a eukaryotic cell.11-24-2011
20100222413Chemically Modified Oligonucleotides for Use in Modulating Micro RNA and Uses Thereof - This invention relates generally to chemically modified oligonuceotides useful for modulating expression of microRNAs and pre-microRNAs. More particularly, the invention relates to single stranded chemically modified oligonuceotides for inhibiting microRNA and pre-microRNA expression and to methods of making and using the modified oligonucleotides. Also included in the invention are compositions and methods for silencing microRNAs in the central nervous system.09-02-2010
20130023577COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 AND VEGF GENES - This invention relates to compositions containing double-stranded ribonucleic acid (dsRNA) in a SNALP formulation, and methods of using the compositions to inhibit the expression of the Eg5 and Vascular Endothelial Growth Factor (VEGF), and methods of using the compositions to treat pathological processes mediated by Eg5 and VEGF expression, such as cancer.01-24-2013
20090281165Antisense oligonucleotide against human acetylcholinesterase (AChE) and uses thereof - The invention relates to an antisense oligonucleotide targeted to the coding region of the human acetylcholinesterase (AChE), which selectively suppresses the AChE-R isoform of the enzyme. The antisense oligonucleotide is intended for use in the treatment and/or prevention of neuromuscular disorders, preferably myasthenia gravis. In addition, it can penetrate the blood-brain barrier (BBB) and destroy AChE-R within central nervous system neurons, while also serving as a carrier to transport molecules across the BBB.11-12-2009
20100168204Therapeutic uses of inhibitors of RTP801L - The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases respiratory conditions and hearing disorders based upon inhibition of the RTP801L gene and/or protein.07-01-2010
20120016012RNAi MOLECULE TARGETING THYMIDYLATE SYNTHASE AND APPLICATION THEREOF - This invention provides a novel RNAi molecule that can significantly potentiate antitumor effects of a 5-FU antitumor agent. The RNAi molecule comprises the nucleotide sequence shown in SEQ ID NO: 2. The invention also provides an antitumor agent comprising such RNAi molecule and a 5-FU antitumor agent.01-19-2012
20110288148TRIGGERED COVALENT PROBES FOR IMAGING AND SILENCING GENETIC EXPRESSION - The present invention relates to the use of cross-linking probes to covalently bind probes to nucleic acid targets. In some embodiments, the probe comprises an initiator region that is able to bind to a first portion of a target nucleic acid, a probe region linked too the initiator region that is able to bind to a second region of the target nucleic acid and that comprises one or more cross-linkers, and a blocking region hybridized to the probe region.11-24-2011
20120022135PHD2 INHIBITION FOR BLOOD VESSEL NORMALIZATION, AND USES THEREOF - A key function of blood vessels, to supply oxygen, is impaired in tumors because of abnormalities in their endothelial lining. PHD proteins serve as oxygen sensors and may regulate oxygen delivery. Therefore the role of endothelial PHD2 in vessel shaping by implanting tumors in PHD201-26-2012
20110130442NUCLEIC ACID CAPABLE OF CONTROLLING DEGRANULATION OF MAST CELL - Provided are a mast cell degranulation control agent, a diagnostic agent or therapeutic agent for a disease resulting from mast cell degranulation control, a method of controlling mast cell degranulation, and a screening method for a mast cell degranulation control agent, all of which involve the use of a nucleic acid and the like. These are useful in the diagnosis or treatment of a disease resulting from mast cell degranulation control.06-02-2011
20110288156Nucleic Acids, Polypetides, and Methods for Modulating Apoptosis - The present invention relates to isolated and/or purified rat apoptosis-specific eucaryotic initiation Factor-5A (eIF-5A) and deoxyhypusine synthase (DHS) nucleic acids and polypeptides. The present invention also relates to methods of modulating apoptosis using apoptosis-specific eIF-5A and DHS, and antisense oligonucleotides and expression vectors of apoptosis-specific and DHS useful in such methods.11-24-2011
20110288157RNAi-Mediated Inhibition of Spleen Tyrosine Kinase-Related Inflammatory Conditions - RNA interference is provided for inhibition of spleen tyrosine kinase (Syk) mRNA expression, in particular, for treating patients having a Syk-related inflammatory condition or at risk of developing a Syk-related inflammatory condition such as allergic conjunctivitis, ocular inflammation, dermatitis, rhinitis, asthma, allergy, or mast-cell disease.11-24-2011
20110288155SIRNA COMPOUNDS AND METHODS OF USE THEREOF - The present application relates to double stranded oligonucleotide inhibitors of target genes, pharmaceutical compositions comprising same and the use of such molecules to treat, inter alia, neurodegenerative disorders including Alzheimer's disease and Amyotrophic Lateral Sclerosis, eye diseases including glaucoma and ION, acute renal failure, hearing loss, acute respiratory distress syndrome and in preventing or treating ischemia-reperfusion injury in organ transplant patients.11-24-2011
20110288153TRANSLATION FACTORS AS ANTI-AGING DRUG TARGETS - In certain embodiments this invention pertains to the discovery that inhibition of the expression and/or activity of eukaryotic initiation factor 4A (eIF4A) inhibits the aging process. Accordingly, in certain embodiments, methods are provided for inhibiting/slowing aging. The methods typically involve administering to a mammal an agent that inhibits the expression and/or activity of eukaryotic initiation factor 4A (eIF4A) in an amount sufficient to inhibit expression or activity of EIF4A, where the agent is not resveratrol.11-24-2011
20110288152PSMA BINDING LIGAND-LINKER CONJUGATES AND METHODS FOR USING - Described herein are prostate specific membrane antigen (PSMA) binding conjugates that are useful for targeting prostate cancer cells. Also described herein are compositions containing them and methods of using the conjugates and compositions. Also described are processes for manufacture of the conjugates and the compositions containing them.11-24-2011
20110288149Method of protecting against heart failure - The present invention relates, in general, to heart failure, and, in particular to a method of reducing the risk of heart failure, particularly in patents with established cardiomyopathy.11-24-2011
20110172289MINOR GROOVE BINDER (MGB)-OLIGONUCLEOTIDE MIRNA ANTAGONISTS - Compositions and methods for inhibiting the actions of non-coding RNAs such as miRNAs and piRNAs are provided. The compositions comprise single or double stranded oligonucleotides conjugated with Minor Groove Binders (“MGBs”). The oligonucleotides can vary in length, can contain nucleotides having one or more modifications, and have regions that are substantially complementary to one or more mature miRNAs or piRNAs.07-14-2011
20100063131PROMPT NUCLEIC ACID DELIVERY CARRIER COMPOSITION - An object of the present invention is to provide a carrier composition for nucleic acid delivery, which can efficiently deliver a nucleic acid into cells when a nucleic acid such as siRNA is administered to animal-derived cells or animals, and also has low toxicity and high safety, and a composition for nucleic acid delivery containing the carrier composition and nucleic acid.03-11-2010
20110301219RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.12-08-2011
20100035969RNAi INHIBITION OF CTGF FOR TREATMENT OF OCULAR DISORDERS - RNA interference is provided for inhibition of connective tissue growth factor mRNA expression in ocular disorders involving CTGF expression. Ocular disorders involving aberrant CTGF expression include glaucoma, macular degeneration, diabetic retinopathy, choroidal neovascularization, proliferative vitreoretinopathy and wound healing. Such disorders are treated by administering interfering RNAs of the present invention.02-11-2010
20100035966METHODS AND COMPOSITIONS FOR REGULATING CELL CYCLE PROGRESSION - In one aspect, a method is provided of inhibiting proliferation of a mammalian cell comprising introducing into said cell an effective amount of at least one at least one small interfering RNA agent (iRNA), wherein said iRNA comprises a nucleotide sequence of at least 15 nucleotides, wherein the nucleotide sequence comprises a seed region consisting of nucleotide positions 1 to 12, wherein position 1 represents the 5′ end of the iRNA nucleotide sequence and wherein said seed region comprises a nucleotide sequence of at least six contiguous nucleotides that is complementary to six contiguous nucleotides within positions 1 to 12 of a nucleotide sequence, wherein position 1 represents the 5″end of the nucleotide sequence, wherein the nucleotide sequence is selected from the group consisting of SEQ ID NO: 1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7 and SEQ ID NO:8. In some embodiments, the method comprises introducing at least one iRNA that inhibits the expression of at least one miR-16 responsive gene selected from TABLE 5 into the mammalian cell.02-11-2010
20100035968MEDIATED CELLULAR DELIVERY OF LNA OLIGONUCLEOTIDES - The present invention relates to novel modified oligomeric compounds and to methods of making and using such compounds. The invention further relates to methods of enhancing the cellular uptake of oligomeric compounds comprising conjugating a metal chelator to those.02-11-2010
20100035964ANTISENSE MODULATION OF CONNECTIVE TISSUE GROWTH FACTOR EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of connective tissue growth factor. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding connective tissue growth factor. Methods of using these compounds for modulation of connective tissue growth factor expression and for treatment of diseases associated with expression of connective tissue growth factor are provided.02-11-2010
20090281164RNA INTERFERENCE MEDIATED INHIBITION OF RESPIRATORY SYNCYTIAL VIRUS (RSV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating sespiratory syncytial virus (RSV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of RSV gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of RSV genes, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules cocktails thereof. The application also relates to methods of treating diseases and conditions associated with RSV gene expression, such as RSV infection, respiratory failure, bronchiolitis and pneumonia, as well as providing dosing regimens and treatment protocols.11-12-2009
20090275637PROTEIN TYROSINE PHOSPHATASE INHIBITORS - The present invention provides compositions and methods for binding and/or modulating enzymatic activity of human protein tyrosine phosphatases such as PTP1B. Additionally, the invention provides methods of identifying and using such nucleic acid ligands.11-05-2009
20090221671MODULATION OF LMW-PTPASE EXPRESSION - Disclosed herein are compounds, compositions and methods for modulating the expression of LMW-PTPase in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of diabetes, insulin resistance, insulin deficiency, hypercholesterolemia, hyperglycemia, dyslipidemia, hyperlipidemia, hypertriglyceridemia, and hyperfattyacidemia. In some embodiments, the diabetes is type II diabetes by administration of antisense compounds targeted to LMW-PTPase.09-03-2009
20120065249ANTISENSE OLIGONUCLEOTIDES FOR TREATING ALLERGY AND NEOPLASTIC CELL PROLIFERATION - Antisense oligonucleotides for treating and/or preventing at least one of asthma, allergy, hypereosinophilia, general inflammation and cancer are provided. The oligonucleotides are directed against nucleic acid sequences coding for a receptor selected from the group consisting of a CCR3 receptor and a common sub-unit of IL-3, IL-5 and GM-CSF receptors.03-15-2012
20100099740METHODS AND COMPOSITIONS FOR RNAI MEDIATED INHIBITION OF GENE EXPRESSION IN MAMMALS - Methods and compositions are provided for modulating, e.g., reducing, expression of a target sequence in mammals and mammalian cells in vivo. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) is introduced into a target cell, e.g., by being administered to a mammal that includes the target cell, e.g., via intravascular injection. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.04-22-2010
20100113558COMPOUNDS AND METHODS - A small nucleic acid molecule that down-regulates expression of Wrap53 gene via RNA interference (RNAi), wherein at least one strand of said small nucleic acid molecule is about 15 to about 30 nucleotides in length; and wherein at least one strand of said small nucleic acid molecule comprises a nucleotide sequence having sufficient complementarity to an RNA of said Wrap53 gene for the small nucleic acid molecule to direct cleavage of said RNA via RNA interference, for use as a medicament.05-06-2010
20110263686ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.10-27-2011
20100113560COMPOSITIONS AND METHODS FOR TREATING HEMATOPOIETIC MALIGNANCIES - Described herein are compositions and methods for the prevention and treatment of hematopoietic malignancies. The compositions are miRNAs and associated nucleic acids.05-06-2010
20100113557METHOD FOR PREVENTION OF TUMOR - The present invention provides a method for inhibiting a tumor, which comprises suppressing the expression of HERC2. In one embodiment of the present invention, the suppression of HERC2 is induced by the expression of BRCA1.05-06-2010
20100113563Method for Treating Pain with a Calmodulin Inhibitor - The present invention relates to the use of Ca05-06-2010
20090312393CANCER THERAPY USING BCL-XL-SPECIFIC SINA - The invention relates to a double-stranded short interfering nucleic acid (siNA) molecule specific to the Bcl-X12-17-2009
20100267811RNAi-RELATED INHIBITION OF TNFa SIGNALING PATHWAY FOR TREATMENT OF OCULAR ANGIOGENESIS - RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enzyme (TACE/ADAM17) mRNA expression. Silencing such TNFα targets, in particular, is useful for treating patients having a TNFα-related condition or at risk of developing a TNFα-related condition, such as ocular angiogenesis, retinal ischemia, and diabetic retinopathy.10-21-2010
20100267803Regulators Of Fat Metabolism As Anti-Cancer Targets - Embodiments of the invention provide methods of identifying agents that reduce or prevent the proliferation of breast cancer cells, or kill them, in particular by interfering with the expression of the transcription factors NR1D1 and PPARγ or the expression of genes whose transcription they activate or the activity of the proteins translated from those transcripts.10-21-2010
20100267809DOUBLE STRANDED NUCLEIC ACID TARGETING LOW COPY PROMOTER-SPECIFIC RNA - The present invention relates to transcriptional gene silencing (TGS) in mammalian, including human, cells that is mediated by small interfering RNA (siRNA) molecules. The present invention also relates to a double stranded nucleic acid that directs methylation of histones associated with target genes that produce low copy promoter-specific RNA. It has been found that siRNAs can be used to direct methylation of histones in mammalian, including human, cells.10-21-2010
20100267804DIFFERENTIAL EXPRESSION OF MICRORNAS IN NONFAILING VERSUS FAILING HUMAN HEARTS - The present invention discloses specific miRNAs as novel biomarkers and therapeutic targets in the treatment of heart failure. Methods of treating or preventing heart failure in a subject by administering mimics or inhibitors of these particular miRNAs are disclosed. A method of diagnosing or prognosing heart failure in a subject by determining the level of expression of one or more miRNAs is also described.10-21-2010
20100267802DELIVERY METHOD - The present invention relates, in general, to RNA silencing and, in particular, to a method of effecting targeted delivery of an RNA silencing moiety using a targeting moiety that binds to a cell surface receptor and mediates internalization of the RNA silencing moiety to be accessible to Dicer. Also provided is a chimeric nucleic acid molecule comprised of a targeting moiety and an RNA silencing moiety, wherein the targeting moiety is an aptamer and the RNA silencing moiety comprises a Dicer substrate.10-21-2010
20120065245REGULATION OF HEMATOPOIETIC STEM CELL FUNCTIONS THROUGH MICRORNAS - The present disclosure relates to regulation of functions of hematopoietic stem cells (HSCs) by delivering of miRNAs, including miR-125b, miR-126, and miR-155, to HSCs. For example, in some embodiments, blood output in a mammal can be increased by administering miR-125b, miR-126, and/or miR-155 oligonucleotides. Also disclosed are methods for promoting hematopoietic stem cell engraftment and method for treating a myeloproliferative disorder.03-15-2012
20110263676SMALL INTERFERING RNA FOR GENE KNOCKDOWN OF THE SUBCUTANEOUS N-METHYL-D-ASPARTATE RECEPTOR NR1 SUBUNIT, AND IT'S APPLICATION ON PHARMACEUTICS - A small interfering RNA for gene knockdown of the N-methyl-D-aspartate receptor NR1 subunit comprises 21 to 25 ribonucleic acids, which are homologous to the RNA sequence of N-methyl-D-aspartate receptor NR1 subunit. A method of using the small interfering RNA, applying the small interfering RNA on subcutaneous tissues temporary interfere with the genetic expression of the NMDA receptor NR1 subunit in hypoderm. A use of the small interfering RNA on pharmaceutics, applying the small interfering RNA manufacture into new analgesic drugs for moderating the inflammatory pain or intolerable chronic pain, especially on clinical chronic pain and burn pain patients. An analgesic drug for skin inflammatory pain comprising: the small interfering RNA and a siRNA acceptable vehicle.10-27-2011
20110263683RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.10-27-2011
20120065243RNA DUPLEXES WITH SINGLE STRANDED PHOSPHOROTHIOATE NUCLEOTIDE REGIONS FOR ADDITIONAL FUNCTIONALITY - The present invention relates to RNAi constructs and their use in gene silencing. RNAi constructs associated with the invention contain a double stranded region connected to a single stranded region of phosphorothioate modified nucleotides.03-15-2012
20100249215Oligomeric Compounds And Compositions For Use In Modulation Of Pri-miRNAs - Compounds, compositions and methods are provided for modulating the levels expression, processing and function of pri-miRNAs. In particular, methods and compounds are provided for the modulation of the levels, expression, processing or function of polycistronic pri-miRNAs. The compositions comprise oligomeric compounds targeted to small non-coding RNAs and pri-miRNAs. Further provided are methods for selectively modulating pri-miRNA levels in a cell. Also provided are methods for identifying oligomeric compounds that result in increase pri-miRNA levels when contacted with a cell.09-30-2010
20100249214MULTIPLEX DICER SUBSTRATE RNA INTERFERENCE MOLECULES HAVING JOINING SEQUENCES - The present invention is based, at least in part, upon the insight that compound DsiRNA agents can be generated using site-specific RNase H-cleavable double stranded nucleic acid double stranded nucleic acid regions to attach, e.g., one DsiRNA moiety to another DsiRNA moiety and/or one DsiRNA moiety to a functional group and/or payload. Because such double stranded nucleic acid joining sequences are site-specifically RNase H-cleavable, the bifunctional molecule is cleaved into DsiRNAs which bear terminal ends that orient dicer cleavage. The detrimental impact of administering a single double stranded nucleic acid RNAi agent of longer than 30-35 nucleotides in length (e.g., provocation of interferon response) can be minimized, as once administered or delivered to a subject or RNase H-containing cell, RNase H cleavage produces a shortened, active DsiRNA agent(s). The invention also provides bifunctional DsiRNA agents that are joined by double stranded DNA extension joining sequences—such bifunctional DsiRNA agents joined by dsDNA sequences do not provoke RNase H cleavage.09-30-2010
20100120892METHODS AND COMPOSITIONS FOR THE INHIBITION OF STAT5 IN PROSTATE CANCER CELLS - The present invention relates to compositions and methods for the treatment of prostate cancer. In certain embodiments, the invention relates to compositions and methods for the inhibition of prostate cancer cell growth, comprising inhibiting the activity of Stat5 in prostate cancer cells.05-13-2010
20100249212Gene Silencing Using mRNA-cDNA Hybrids - The present invention provides novel compositions and methods for suppressing the expression of a targeted gene using mRNA-cDNA duplexes. The invention further provides novel methods and compositions for generating amplified mRNA-cDNA hybrids, whose quantity is high enough to be used for the invention's gene silencing transfection. This improved RNA-polymerase chain reaction method uses thermocycling steps of promoter-linked double-stranded cDNA or RNA synthesis, in vitro transcription and then reverse transcription to amplify the amount of mRNA-cDNA hybrids up to two thousand folds within one round of the above procedure.09-30-2010
20100249211N-Substituted-Aminomethylene Bridged Bicyclic Nucleic Acid Analogs - Provided herein are bicyeMc nucleosides comprising a substituted amino group in the bridge, oligomeric compounds having at least one of these bicyclic nucleosides and methods of using the oligomeric compounds. The bicyclic nucleosides comprising a substituted amino group in the bridge are useful for enhancing properties of oligomeric compounds including nuclease resistance, in certain embodiments, the oligomeric compounds hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.09-30-2010
20100249209GENE INVOLVED IN IMMORTALIZATION OF HUMAN CANCER CELL AND USE THEREOF - The present invention provides a gene related to cancer cell immortalization (an immortalization determining gene) and a process that is useful for selective cancer treatment targeting a cancer cell having the gene. The present invention determines an immortalized cancer cell using a polynucleotide having a base sequence of at least 15 bases that specifically hybridizes with a continuous base sequence of at least 15 bases within any one of abase sequences represented by SEQ ID Nos. 1 to 13. In the foregoing process, the polynucleotide is used as a primer or probe for detecting an immortalization determining gene that exhibits high expression specifically in an immortalized cancer cell.09-30-2010
20120142754MODULATION OF TIMP1 AND TIMP2 EXPRESSION - Provided herein are compositions, methods and kits for modulating expression of target genes, particularly of tissue inhibitor of metalloproteinase 1 and of tissue inhibitor of metalloproteinase 2 (TIMP1 and TIMP2, respectively). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding TIMP1 and TIMP2, for example, the gene encoding human TIMP1 and TIMP2. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with TIMP1 and TIMP2 including fibrotic diseases and disorders including liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis.06-07-2012
20110172291RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS - The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.07-14-2011
20120035246SINGLE-STRANDED NUCLEIC ACID MOLECULE HAVING NITROGEN-CONTAINING ALICYCLIC SKELETON - Provided is a novel nucleic acid molecule that can be produced easily and efficiently and can inhibit the expression of a gene. The nucleic acid molecule is a single-stranded nucleic acid molecule including an expression inhibitory sequence that inhibits expression of a target gene. The single-stranded nucleic acid molecule includes: a region (X); a linker region (Lx); and a region (Xc). The linker region (Lx) is linked between the regions (Xc) and (Xc). The region (Xc) is complementary to the region (X). At least one of the regions (X) and (Xc) includes the expression inhibitory sequence. The linker region (Lx) has a non-nucleotide structure including at least one of a pyrrolidine skeleton and a piperidine skeleton. According to this single-stranded nucleic acid molecule, it is possible to inhibit the expression of the target gene.02-09-2012
20120035244PARP1 TARGETED THERAPY - The present invention relates to compositions and methods for cancer therapy, including but not limited to, targeted inhibition of cancer markers. In particular, the present invention relates to PARP1 proteins and nucleic acids as clinical and research targets for cancer.02-09-2012
20120035243DUAL TARGETING OF MIR-208 AND MIR-499 IN THE TREATMENT OF CARDIAC DISORDERS - The present invention provides a method of treating or preventing cardiac disorders in a subject in need thereof by inhibiting the expression or function of both miR-499 and miR-208 in the heart cells of the subject. In particular, specific protocols for administering inhibitors of the two miRNAs that achieve efficient, long-term suppression are disclosed. In addition, the invention provides a method for treating or preventing musculoskeletal disorders in a subject in need thereof by increasing the expression or activity of both miR-208 and miR-499 in skeletal muscle cells of the subject.02-09-2012
20120035241USE OF INHIBITORS OF PLAC8 ACTIVITY FOR THE MODULATION OF ADIPOGENESIS - The present invention concerns Plac8, a new target involved in adipogenesis modulation. Using a siRNA approach, the inventors demonstrated that decrease in Plac8 activity in preadipocytes and adipose tissue induces a decrease in adipogenesis. Thus, the present invention relates to modulators of Plac8 activity as well screening test for identification of modulators as of the activity of this target, and their use, especially in pharmaceutical composition, to modulate adipogenesis and thus treat obesity and related disorders.02-09-2012
20120035240CONSERVED HBV AND HCV SEQUENCES USEFUL FOR GENE SILENCING - Conserved consensus sequences from known hepatitis B virus strains and known hepatitis C virus strains, which are useful in inhibiting the expression of the viruses in mammalian cells, are provided. These sequences are useful to silence the genes of HBV and HCV, thereby providing therapeutic utility against HBV and HCV viral infection in humans.02-09-2012
20090253772RNA INTERFERENCE MEDIATED INHIBITION OF CXCR4 GENE EXPRESSION USING SHORT INTERFERING NUCELEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating chemokine receptor (CXCR) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of CXCR gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of CXCR genes such as CXCR4 and CXCR7A.10-08-2009
20110021608METHODS FOR NUCLEIC ACID TRANSFER INTO CELLS - The present invention provides methods for increasing the transfer of nucleic acids into cells. In particular, the present invention provides for the use of inhibitors of HDAC6, a cytoplasmic histone deacetylase present in mammalian cells by, for example, small molecules or siRNA treatment, in increasing gene transfer and/or expression in cells in vitro and in vivo for research and gene therapy applications.01-27-2011
20100286235METHODS FOR INCREASING IN VIVO EFFICACY OF OLIGONUCLEOTIDES AND INHIBITING INFLAMMATION IN MAMMALS - The invention relates to the use of nucleotide substitutes for increasing the in vivo efficacy of nucleic acid molecules and also for inhibiting inflammation in mammals. More particularly, the present invention relates to the use of 2′6′ diaminopurine (DAP) and analogs thereof per se in anti-inflammatory compositions, and also for preparing nucleic acid molecules having an increased in vivo physiological efficiency and a reduced toxicity as compared to conventional oligos. The invention is particularly useful for the preparation of antisense oligonucleotides for treating pulmonary/respiratory diseases such as cystic fibrosis, asthma, chronic bronchitis, chronic obstructive lung disease, eosinophilic bronchitis, allergies, allergic rhinitis, pulmonary fibrosis, adult respiratory distress syndrome, sinusitis, respiratory syncytial virus or other viral respiratory tract infection and cancer.11-11-2010
20100087508Compositions and Methods for Inhibiting Expression of Eg5 and VEGF Genes - This invention relates to compositions containing double-stranded ribonucleic acid (dsRNA) in a SNALP formulation, and methods of using the compositions to inhibit the expression of the Eg5 and Vascular Endothelial Growth Factor (VEGF), and methods of using the compositions to treat pathological processes mediated by Eg5 and VEGF expression, such as cancer.04-08-2010
20120108650MICRO RNA MARKERS AND METHODS RELATED THERETO - The present invention provides methods of diagnosis of Alzheimer's disease including assessing the levels of certain microRNAs in a subject and comparing these to levels in subjects not exhibiting the disease. The identified measurements provide input for improved diagnoses of Alzheimer's disease as compared to certain other forms of dementias, which allows more effective treatment regimens.05-03-2012
20120108649METHODS AND COMPOSITIONS TO PROTECT AQUATIC INVERTEBRATES FROM DISEASE - Compositions and methods of protecting aquatic invertebrates from disease is shown. In one embodiment, dsRNA or antisense RNA to a nucleic acid molecule of the disease-causing microorganism is prepared and delivered to the animal. In another embodiment, a nucleic acid molecule of the disease-causing microorganism is delivered to the animal. In another embodiment, the RNA or nucleic acid molecule is delivered to the animal by replicon particle. In a further embodiment, the protective molecule is delivered to the digestive tract of the animal. Protection from disease is obtained.05-03-2012
20090306186NOVEL TISSUE PROTECTIVE ERYTHROPOIETIN RECEPTOR (NEPOR) AND METHODS OF USE - There is disclosed a molecular composition(s) of a novel tissue protective erythropoietin (EPO) binding receptor protein complex, termed NEPOR. Presence of NEPOR components on a tumour allows EPO to impinge on the survival of associated cells thereby enhancing tumour progression and negatively effecting patient survival. Presence of NEPOR represents a prognostic biomarker for poorer patient outcome. Thus, methods are provided for stratifying patients having a tumour as suitable (i.e. NEPOR not present) or non-suitable (i.e., NEPOR present) for EPO treatment, comprising: (a) isolating a tissue sample from an individual who is receiving or is a candidate for receiving erythropoietin, (b) determining the level of expression of the NEPOR gene(s) (mRNA) and/or the presence of the NEPOR gene product (protein) from the isolated tissue, and (c) correlating the presence of an NEPOR gene expression product or the presence of NEPOR protein to a physiological response to the treatment with erythropoietin. Furthermore, by disclosing the molecular compositions of NEPOR species, there are disclosed methods for rationally identifying/designing NEPOR modulating therapeutics. Methods also are provided for treating neurological insults such as stroke (via enhancement of NEPOR activity) and cancer (via down-regulation of cyto-protective signaling from NEPOR).12-10-2009
20090306178CONJUGATED DOUBLE STRAND COMPOSITIONS FOR USE IN GENE MODULATION - The present invention provides conjugated double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and/or sugar modified nucleosides. More particularly, the present compositions comprise a linked conjugate group on one strand and a non hybridizing region of 2′-modified nucleosides on the other strand. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.12-10-2009
20100099743RNA INTERFERENCE MEDIATED INHIBITION OF CHECKPOINT KINASE-1 (CHK-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating checkpoint kinase (e.g., checkpoint kinase-1 or CHK-1) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of checkpoint kinase gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of checkpoint kinase genes.04-22-2010
20100081705METHODS FOR SLOWING FAMILIAL ALS DISEASE PROGRESSION - Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS.04-01-2010
20100081704MODULATOR COMPOUNDS OF THE DRUG RESISTANCE IN EPITHELIAL TUMOUR CELLS - The use of compounds is described which are capable of functionally blocking at least one of the genes chosen from the group composed of EphA1, EphA2, EphA8, EphB2, CSF1R, VEGFR2, RAMP2, RAMP3, CLRN1, MAPK4, PIK3C2A, PIK3CG, GSK3alpha, GSK3beta, IRAK3, DAPK1, JAK1, PIM1, TRB3, BTG1, LATS1, LIMK2, MYLK, PAK1, PAK2, CDC2, BTK, PNRC2, NCOA4, NR2C1, TPR, RBBP8, TRPC7, FXYD1, ERN1, PRSS16, RPS3, CCL23 and SERPINE1, for the manufacture of a medicament destined to diminish the resistance to chemotherapeutic drugs in the therapeutic treatment of epithelial tumour pathologies. Also described is a method for the determination of the drug resistance in tumour cells, as well as a method for the identification of tumour stem cells.04-01-2010
20090292003CELL PENETRATING PEPTIDES FOR INTRACELLULAR DELIVERY OF MOLECULES - The present invention concerns cell-penetrating peptides which comprise an amino acid sequence consisting of GLX11-26-2009
20100125099THERAPEUTIC INTERVENTION IN A GENETIC DISEASE IN AN INDIVIDUAL BY MODIFYING EXPRESSION OF AN ABERRANTLY OR ABNORMALLY EXPRESSED GENE - The present invention provides means and methods for alleviating genetic disease. A genetic defect that has a phenotype in differentiated cells can lead to defects in precursor cells thereof. These so-called secondary defects contribute to the overall disease of the individual. In the present invention, genetic intervention with the aim to alleviate symptoms of genetic disease is directed toward the primary genetic defect in the differentiated cell and the secondary defect in the precursor cell.05-20-2010
20100099742Sensitizing Cells for Apoptosis by Selectively Blocking Cytokines - The invention refers to the use of a cytokine antagonist which modulates the expression and/or the function of a cytokine, particularly a Th2 helper cell cytokine, in a cell and causes the down-regulation of anti-apoptotic proteins in said cell through the cytokine modulation for sensitizing cells for apoptosis. In particular, the cells that can be treated with the cytokine antagonists are drug-resistant cancer cells which fail to undergo apoptosis.04-22-2010
20100099741Molecular Targets for Treatment of Learning and Memory Dysfunction - Described herein are methods for identification of alternative safe molecular targets and novel regulators in complex molecular networks using a novel fault diagnosis engineering approach. For example, in this invention we claim new molecular targets that could be effectively targeted for changing the activity of CREB, and therefore for treatment of learning and memory related disorders. Learning and memory dysfunction is a major clinical manifestation of a number of human disorders, such as Alzheimer Disease (AD), schizophrenia, dementias, autism, etc. More specifically, we claim that composition and compounds that can target the activity of L AND/OR P/Q-, N-type calcium channels, Gαi, Gβγ, PP2A and CaMKII and IV are effective therapeutics for the treatment of disorders manifested by learning and memory dysfunction.04-22-2010
20100280102DOUBLE-STRANDED RIBONUCLEIC ACID WITH INCREASED EFFECTIVENESS IN AN ORGANISM - The present invention relates to a method for the targeted selection of a double-stranded ribonucleic acid (dsRNA) consisting of two single strands that exhibits increased effectiveness in inhibiting the expression of a target gene by means of RNA interference, wherein at least end of the dsRNA comprises a nucleotide overhang of 1 to 4 unpaired nucleotides in length; wherein the unpaired nucleotide adjacent to the terminal nucleotide pair comprises a purine base; and wherein the terminal nucleotide pair on both ends of the dsRNA is a G-C pair, or at least two of the last four consecutive terminal nucleotide pairs are G-C pairs.11-04-2010
20090253771INHIBITION OF GLIOTOXIN - The present invention relates to the inhibition of the interaction between Gliotoxin (GT) and its intracellular target for the prevention and/or treatment of fungal infections. Further, novel methods and systems for identifying antifungal agents are disclosed.10-08-2009
20110172290Isoforms of eIF-5A: Senescence-induced eIF5A; Wounding-induced elF-5A; Growth elF-5A; and DHS - The present invention relates to unique isoforms of eukaryotic initiation Factor 5A (“eIF-5A”): senescence-induced eIF-5A; wounding-induced eIF-5A; and growth eIF-5A, as well as polynucleotides that encode these three factors. The present invention also relates to methods involving modulating the expression of these factors. The present invention also relates to deoxyhypusine synthase (“DHS”), polynucleotides that encode DHS, and methods involving modulating the expression of DHS.07-14-2011
20110172287COMPOSITIONS AND METHODS FOR INHIBITING TUMOR GROWTH AND METASTASIS - Disclosed are compositions and methods useful in the reduction of p11 protein activity in cancer cells. P11 protein is demonstrated to affect plasmin production and activity, MMP activity, plasminogen activation, antiangiogenic plasmin fragment production, cell invasion, tumor development and metastasis. Compositions that modulate levels of p11 either up or down are demonstrated to be effective in reducing tumor development. Also disclosed are cancer treatment methods that employ compositions that modulate p11 activity and clonal cell lines and assays useful for the identification of compositions that modulate p11 activity.07-14-2011
20110172286CD44 SPLICE VARIANTS IN NEURODEGENERATIVE DISEASES - There is provided a method of treating or preventing a neurodegenerative disease, which includes administration of a composition that includes a reagent capable of modulating expression of ribonucleic acid (RNA) encoded by a nucleic acid, wherein the nucleic acid is selected from a group that includes a contiguous nucleotide sequence being at least 90% homologous to at least 20 nucleotides of: SEQ ID NO: 1, SEQ ID NO: 3, SEQ ID NO: 5, SEQ ID NO: 7, or any combination thereof. There is further provided a method of treating or preventing a neurodegenerative disease, which includes administration of a composition that includes a reagent capable of modulating expression and/or activity of a polypeptide, wherein the sequence of the polypeptide is selected from a group that includes a contiguous amino acid sequence being at least 90% homologous to at least 10 amino acid of: SEQ ID NO: 2, SEQ ID NO: 4, SEQ ID NO: 6, SEQ ID NO: 8, or any combination thereof.07-14-2011
20100125100GOLD NANOROD-siRNA COMPLEXES AND METHODS OF USING SAME - Provided are methods and compositions for inhibiting expression of one or more target genes. The compositions contain RNA polynucleotides that can inhibit expression of a target gene via RNA interference (RNAi) electrostatically complexed with surface functionalized gold nanorods (GNRs). The RNA polynucleotides are not covalently bound to the surface functionalized GNRs. The method involves inhibiting expression of a target gene in an individual. The method is performed by administering to the individual an effective amount of a composition containing surface functionalized GNRs electrostatically complexed with RNA polynucleotides, such as siRNA, that can inhibit expression of the target gene via RNAi. The siRNA is not covalently bound to the surface functionalized GNRs.05-20-2010
20090275631THERAPEUTIC ANTISENSE OLIGONUCLEOTIDE COMPOSITION FOR THE TREATMENT OF INFLAMMATORY BOWEL DISEASE - Disclosed herein is a method for the sustained amelioration and/or treatment of ulcerative colitis comprising rectal administration of a compound comprising an antisense oligonucleotide having the sequence 5′-GCCCAAGCTGGCATCCGTCA-3′, ISIS 2302. The method results in a decrease in the indications of ulcerative colitis for an extended period (greater than 90 days) after the conclusion of the administration of the composition. The composition is well tolerated and systemic exposure is minimal.11-05-2009
20090281167COMPOSITIONS AND METHODS RELATED TO MIRNA MODULATION OF NEOVASCULARIZATION OR ANGIOGENESIS - The present invention concerns methods and compositions for diagnosing and/or treating vascular diseases including cancer, cardiac diseases, vascular diseases of the eye, and inflammatory diseases. The methods involve measuring the levels of one or multiple miRNAs in patient samples and using the test results to diagnose and/or predict an optimal treatment regimen for the patient. Compositions described in the invention include nucleic acids that function as miRNAs or miRNA inhibitors that can be introduced to a patient to reduce or increase vascularization as needed.11-12-2009
20090292007Inhibition of TACE or amphiregulin for the Modulation of EGF Receptor Signal Transactivation - The present invention relates to the modulation of transactivation of receptor tyrosine kinases by G protein or G protein-coupled receptor (GPCR) mediated signal transduction in a cell or an organism comprising inhibiting the activity of the metalloprotease TACE/ADAM17 and/or the activity of the receptor tyrosine kinase ligand amphiregulin.11-26-2009
20110172292ANTIDOTE OLIGOMERS - The present invention relates to antidote oligomeric compounds (oligomers), which target nucleotide based therapeutics in vivo, thereby providing a method of controlling the bioavailability and therefore the therapeutic activity and/or side effects of nucleotide based therapeutic in vivo.07-14-2011
20110172288Compositions and Methods for the Identification, Assessment, Prevention and Therapy of Thymic Lymphoma or Hamartomatous Tumours - The subject matter relates to newly discovered nucleic acid molecules and proteins associated with thymic lymphoma and hamartomatous tumors. Compositions and methods for detecting, characterizing, preventing, and treating human thymic lymphoma and hamartomatous tumors are provided.07-14-2011
20090275636PICORNAVIRUS AND USES THEREOF - The invention is directed to a a clade of newly isolated and identified picornaviruses associated with respiratory infection, and isolated nucleic acids sequences and peptides thereof. The invention also relates to antibodies against antigens derived from the picornavirus. The invention also relates to iRNAs which target nucleic acid sequences of the picornavirus. The invention is related to methods for detecting the presence or absence of picornavirus in a subject. The invention is also related to immunogenic compositions for inducing an immune response against picornavirus in a subject.11-05-2009
20090275638Compositions and Methods for Inhibiting Expression of XBP-1 Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting X-Box Protein 1 (XBP-1), and methods of using the dsRNA to inhibit expression of XBP-1.11-05-2009
20090275635SCREENING METHOD - The invention provides a method of modulating Wnt signalling comprising modulating Trabid activity. Preferably modulating Trabid activity comprises inhibiting; Trabid activity. The invention also provides a method of reducing TCF transcription, said method comprising reducing Trabid activity. A method for identifying a-modulator of Trabid said method comprising; providing a Trabid substrate comprising a detectable moiety coupled to a tag moiety by ubiquitin; immobilising first and second portions of said substrate; adding a candidate modulator to the first said portion; contacting first and second portions with Trabid; incubating to allow Trabid action, assaying cleavage of ubiquitin by separation of tag from detectable moiety, wherein separation of an amount of detectable moiety from said first portion which is different from the amount of detectable moiety separated from said second portion identifies said candidate as a modulator of Trabid. The invention provides uses of Trabid and of Trabid inhibitors as-medicaments.11-05-2009
20090275639USP47 INHIBTORS AND METHODS TO INDUCE APOPTOSIS - The present invention relates to USP47 (ubiquitin specific protease 47) inhibitors and methods for inducing apoptosis or cell death in a target cell. In certain embodiments, the invention relates to methods and kits to screen for related agents that induce apoptosis. Additionally, the invention relates to assays for screening compounds capable of acting as USP47 inhibitors.11-05-2009
20090275641RNAi-Mediated Inhibition of RHO Kinase for Treatment of Ocular Disorders - RNA interference is provided for inhibition of Rho kinase mRNA expression for treating patients with ocular disorders, particularly for treating intraocular pressure, ocular hypertension and glaucoma. Rho kinase mRNA targets include mRNA for ROCK1 and ROCK2.11-05-2009
20090275640siRNA targeting inner centromere protein antigens (INCENP) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for INCENP.11-05-2009
20090281163REGULATORY ELEMENTS THAT MEDIATE RETINAL CELL-SPECIFIC GENE EXPRESSION - Isolated nucleic acid molecules including regulatory elements that direct retinal-cell specific expression are provided. Methods for treating or preventing retinal disorders in a subject are also provided.11-12-2009
20090286849MODULATION OF INSULIN LIKE GROWTH FACTOR I RECEPTOR EXPRESSION - The present invention provides compositions and methods for modulating the expression of growth factor gene. In particular, this invention relates to compounds, particularly oligonucleotide compounds, which, in preferred embodiments, hybridize with nucleic acid molecules encoding the Insulin Like Growth Factor I receptor (IGF-I receptor or IGF-IR) and in particular human IGF-IR. Such compounds are exemplified herein to modulate proliferation which is relevant to the treatment of proliferative and inflammatory skin disorders and cancer. It will be understood, however, that the compounds can be used for any other condition in which the IGF-IR is involved including inflammatory condition.11-19-2009
20090286850Inhibition of EMT induction in tumor cells by anti-cancer agents - The present invention provides methods of identifying an agents that inhibit tumor cells from undergoing an epithelial to mesenchymal transition, impair tumor cell mobility, and thus inhibit tumorigenicity. The present invention also provides compositions comprising said agents, and methods for their preparation and use. The present invention also provides methods for inhibiting tumor cells in a patient from undergoing an epithelial to mesenchymal transition by administration of inhibitors of PAK2 kinase, that optionally also inhibit PAK1 kinase. Such methods may be employed in combination with other anti-cancer agents such as EGFR or IGF-1R kinase inhibitors.11-19-2009
20090292005GALACTOSE DERIVATIVE, DRUG CARRIER AND MEDICINAL COMPOSITION - The object of the present invention is to provide a novel and useful galactose derivative, which is a component of a drug carrier by which a medicine can be efficiently transferred into the liver, a drug carrier comprising the derivative, and a pharmaceutical composition comprising the drug carrier and a medicine.11-26-2009
20090292009MODULATION OF STAT 6 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of STAT 6. The compositions comprise oligonucleotides, targeted to nucleic acid encoding STAT 6. Methods of using these compounds for modulation of STAT 6 expression and for diagnosis and treatment of disease associated with expression of STAT 6 are provided.11-26-2009
20090292008Compositions and Methods for Treatment of Prostate and Other Cancers - Therapeutic agents which target heat shock protein (hsp) 27 in vivo are used to provide treatment to individuals, particularly human individuals, suffering from prostate cancer and other cancers that overexpress hsp27. A therapeutic agent, for example an antisense oligonucleotide or RNAi nucleotide inhibitor with sequence specificity for hsp27 mRNA, for example human hsp27 mRNA, is administered to an individual suffering from prostate cancer or some other cancer expressing elevated levels of hsp27 in a therapeutically effective amount. The therapeutic agent is suitably formulated into a pharmaceutical composition which includes a pharmaceutically acceptable carrier, and packaged in dosage unit form. A preferred dosage unit form is an injectable dosage unit form.11-26-2009
20090292006COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF DGAT2 - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.11-26-2009
20110172293Methods and Compositions for Modulating Angiogenesis - The present invention provides compositions comprising antisense nucleic acids that reduce miR-126 levels in an endothelial cell. The present invention provides compositions comprising a target protector nucleic acid. The present invention provides methods of modulating angiogenesis in an individual, the methods generally involving administering to the individual an effective amount of an agent that increases or that decreases the level of miR-126 in endothelial cells of the individual.07-14-2011
20110172294CYCLOHEXENYL NUCLEIC ACIDS ANALOGS - The present disclosure describes cyclohexenyl nucleic acid analogs, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, cyclo-hexenyl nucleic acid analogs are provided, having one or more chiral substituents, that are expected to be useful for enhancing properties of oligomeric compounds including nuclease resistance and binding affinity. In some embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.07-14-2011
20090298911SIRNA HAVING ANTIVIRAL ACTIVITY AGAINST NONPOLIO ENTEROVIRUS - The present invention relates to an siRNA (small interfering RNA) having antiviral activity against nonpolio enteroviruses, and a pharmaceutical composition comprising same as an active ingredient for preventing and treating diseases caused by nonpolio enterovirus infection.12-03-2009
20090298916Pharmaceutical compositions for treatment of microRNA related diseases - The present invention provides compositions and methods of treatment of diseases that are sensitive to drugs that downregulate the function of microRNA's, mRNA, non-coding RNA, or viral genomes. In particular, it has been discovered that a very long term effect of an anti microRNA oligonucleotide may be obtained when administered to a primate. Therefore, the present invention relate to pharmaceutical compositions and methods for treatment of primates, including humans wherein the compositions are administered with a long time interval.12-03-2009
20090298909Multiple RNA Polymerase III Promoter Expression Constructs - Expression constructs comprising at least two different RNA polymerase III promoters, wherein each promoter is operably linked to a nucleic acid sequence encoding an RNA effector molecule, are disclosed herein. Further provided are expression constructs comprising multiple polymerase III promoters operably linked to sequences encoding short hairpin RNA molecules, which may comprise single and/or multiple fingers. The provided constructs are useful for in vivo delivery of RNA molecules effective in gene silencing, including of viral genes including HBV and HCV.12-03-2009
20110201669METHODS AND COMPOSITIONS FOR THE DIAGNOSIS AND TREATMENT OF CANCER - The present invention relates to the diagnosis and treatment of cancer, and in particular breast cancer. Specifically, in some embodiments the invention relates to methods of diagnosing cancer, and in particular breast cancer, using an antibody specific for a gene product that localizes selectively to the endoplasmic reticulum of the cancer cell(s). In some embodiments, the invention relates to methods of treating cancer, and in particular breast cancer, by administering a composition comprising an RNA interference sequence (e.g., shRNA, RNAi and/or siRNA molecule) characterized by an ability to inhibit an mRNA molecule, which mRNA molecule is encoded by the C43 gene (SEQ ID NO: 1). The invention additionally relates to methods for detecting cancer cells by detecting reduced methylation of the C43 promoter, and methods for reducing cancer metastasis by using demethylation inhibitors that result in increased methylation of the C43 promoter. The invention additionally relates to an in vitro 3-dimensional assay for detecting migrating cells, identifying test agents and/or nucleotide sequences that alter cell migration.08-18-2011
20090203766vWF aptamer formulations and methods for use - The invention relates to the formulation, dosing, administration and use of an aptamer antagonist therapeutic that binds to von Willebrand Factor.08-13-2009
20130217750Cell Growth Inhibitor and Screening Method Thereof - An object is to provide a cell growth inhibitor also effective for androgen-independent prostate cancer. The present invention provides a cell growth inhibitor having, as an active ingredient, an expression inhibitor or function inhibitor of PSF.08-22-2013
20110207799Compositions for Targeted Delivery of siRNA - The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to hepatocytes in vivo. Targeted RNAi polynucleotides are administered together with co-targeted delivery polymers. Delivery polymers provide membrane penetration function for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness to the delivery polymers.08-25-2011
20120295951GENETIC CHANGES IN ATM AND ATR/CHEK1 AS PROGNOSTIC INDICATORS IN CANCER - The present invention relates to the discovery that, in human cancer, an 11q deletion of ATM together with an increase in ATR and CHEK1 expression correlates with resistance to ionizing radiation which could be overcome by inhibition of the ATR/CHEK1 pathway. It provides for methods of identifying patients unlikely to exhibit an adequate response to radiation therapy and/or chemotherapy who may benefit from ATR/CHEK1 pathway inhibition, as well as methods of treating said patients.11-22-2012
20120295950RBP4 IN INSULIN SENSITIVITY/RESISTANCE, DIABETES, AND OBESITY - Methods for screening molecules that modulate the activity of Retinol Binding Protein 4 (RBP4) and their use in treatment of insulin resistance are described. Also described are methods of diagnosing insulin resistance and related conditions by detecting modulation of RBP4 activity.11-22-2012
20120295949MICRORNA-BASED METHODS AND COMPOSITIONS FOR THE DIAGNOSIS, PROGNOSIS AND TREATMENT OF TUMOR INVOLVING CHROMOSOMAL REARRANGEMENTS - Provided are novel methods and compositions for the diagnosis, prognosis and treatment of tumor involving a chromosomal rearrangement, in particular a tumor or neoplasia of the thyroid gland. In addition, methods of identifying anti-tumor agents are described.11-22-2012
20120295953TREATMENT OF TUMOR PROTEIN 63 (P63) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO P63 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Tumor Protein 63 (p63), in particular, by targeting natural antisense polynucleotides of Tumor Protein 63 (p63). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of p63.11-22-2012
20110207798METHOD FOR TREATING BREAST CANCER USING ADENINE NUCLEOTIDE TRANSLOCATOR 2 (ANT2) SIRNA OR ANT2 SHRNA - The present invention relates to adenine nucleotide translocator 2 (ANT2) siRNA (small interfering RNA) or ANT2 shRNA (short hairpin RNA) suppressing the expression of ANT2 gene expression and anticancer agent containing the same. Furthermore, the present invention relates to methods for treating breast cancers or stem cells of a breast cancer by treating the same with ANT2 siRNA or ANT2 shRNA. In addition, the invention provides a method for inhibiting metastasis of breast cancer cells with ANT2 siRNA or ANT2 shRNA.08-25-2011
20110207796ALPHA-SYNUCLEIN KINASE - Agents and methods for treatment of diseases associated with Lewy body diseases (LBDs) in the brain of a patient are provided. Preferred agents include inhibitors of PLK2 kinase.08-25-2011
20100280101METHOD OF INHIBITING CANCER CELL PROLIFERATION, PROLIFERATION INHIBITOR AND SCREENING METHOD - The present invention provides a method of inhibiting cancer cell proliferation by suppressing a function of ZNF143, a cancer cell proliferation inhibitor containing as an active ingredient a substance capable of inhibiting a function of ZNF143, like a ZNF143-specific siRNA, a prophylactic and/or therapeutic drug for cancer, a method of detecting cancer cells, a diagnostic reagent for cancer, a vector and transformant cell incorporating the vector, and a screening method for a substance possessing cancer cell proliferation inhibitory activity with the amount of inhibition of the binding of ZNF143 protein as an index.11-04-2010
20100286232MICRORNA EXPRESSION PROFILE ASSOCIATED WITH PANCREATIC CANCER - Methods are provided for diagnosing whether a subject has, or is at risk of developing, pancreatic cancer. The methods include measuring the level of at least one miR gene product in a biological sample derived from the subject's pancreas. An alteration in the level of the miR gene product in the biological sample as compared to the level of a corresponding miR gene product in a control sample, is indicative of the subject either having, or being at risk for developing, pancreatic cancer.11-11-2010
20100137405RNA Interference Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase (PDE4B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL-15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP-2, MMP-3, MMP-9 and MMP-12. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC) including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.06-03-2010
20100137409Compositions and Methods for the Treatment of Diseases Associated with Aberrant Cilia Assembly and Regulation - Compositions and methods are provided for identifying agents which have efficacy for the treatment of disorders related to aberrant cilial structure and function, including polycystic kidney disease.06-03-2010
20100137410Oncogenic ALL-1 Fusion Proteins for Targeting Drosha-Mediated MicroRNA Processing - Disclosed are compositions and methods for reducing the proliferation of ALL cancer cells through targeted interactions with ALL1 fusion proteins.06-03-2010
20100099739Compositions and Methods for Gene Silencing - Compositions and methods for modulating the expression of a protein of interest are provided.04-22-2010
20100280097COMPOSITIONS COMPRISING HIF-1 ALPHA SIRNA AND METHODS OF USE THEREOF - The present invention provides nucleic acid molecules that inhibit HIF-1α expression. Methods of using the nucleic acid molecules are also provided.11-04-2010
20100280103Methods and compositions for acid phosphatase-1 gene inhibition - The ACP1 *A allele provides a means for diagnosing susceptibility of a human subject to hyperlipidemia, especially hyperlipidemia associated with metabolic syndrome, a means for treating, or preventing the onset of, hyperlipidemia and metabolic syndrome, and a means for screening and identifying drugs suitable for use in treating or preventing hyperlipidemia, especially hyperlipidemia associated with metabolic syndrome. Diagnostic kits are also provided.11-04-2010
20100280099Combination Treatment For The Treatment of Hepatitis C Virus Infection - The present invention relate to the use of a combination of an inhibitor of miR-122 and an inhibitor of VLDL assembly, for the treatment of HCV, hyperlipidemia and hypercholesterolemia.11-04-2010
20100280096TREATMENT OF INFLUENZA - The present invention provides a double-stranded RNA which inhibits replication of influenza B viruses by RNA interference, in which the double-stranded RNA comprises an RNA having 19 to 25 nucleotides homologous with a part of an mRNA transcribed from a genomic RNA of the influenza B viruses and an antisense RNA thereof.11-04-2010
20100280094COMPOSITIONS AND METHODS TO TREAT MUSCULAR & CARDIOVASCULAR DISORDERS - The present invention relates to a novel microRNA, mir-208-2, implicated in muscular and cardiovascular disorders. The present invention also relates to oligonucleotide therapeutic agents (antisense oligonucleotides and/or double stranded oligonucleotides such as dsRNA) and their use in the treatment of muscular and cardiovascular disorders resulting from dysregulation of mir-208-2.11-04-2010
20100273860MODULATION OF APOLIPOPROTEIN (a) EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of apolipoprotein(a). The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein(a). Methods of using these compounds for modulation of apolipoprotein(a) expression and for diagnosis and treatment of disease associated with expression of apolipoprotein(a) are provided.10-28-2010
20130217755ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.08-22-2013
20130217751POLYNUCLEOTIDES FOR REDUCING RESPIRATORY SYNCYTIAL VIRUS GENE EXPRESSION - This invention pertains to polynucleotides, such as small interfering RNA (siRNA), useful for reducing the expression of respiratory syncytial virus (RSV) genes within a subject; and methods for treating a patient suffering from, or at risk of developing, an RSV infection by administering such polynucleotides to the subject.08-22-2013
20120046344TREATMENT OF LIPID TRANSPORT AND METABOLISM GENE RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO A LIPID TRANSPORT AND METABOLISM GENE - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Lipid transport and metabolism gene, in particular, by targeting natural antisense polynucleotides of a Lipid transport and metabolism gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of a Lipid transport and metabolism genes.02-23-2012
20120295959TREATMENT OF RNASE H1 RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO RNASE H1 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of RNAse H1, in particular, by targeting natural antisense polynucleotides of RNAse H1. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of RNase H1.11-22-2012
20120295956PREVENTION OF TISSUE ISCHEMIA AND RELATED METHODS - Provided herein are methods for preventing, ameliorating, and/or reducing tissue ischemia and/or tissue damage due to ischemia, increasing blood vessel diameter, blood flow and tissue perfusion in the presence of vascular disease including peripheral vascular disease, atherosclerotic vascular disease, coronary artery disease, stroke and influencing other conditions, by suppressing CD47 and/or blocking TSP1 and/or CD47 activity or interaction. Influencing the interaction of CD47-TSP1 in blood vessels allows for control of blood vessel diameter and blood flow, and permits modification of blood pressure and cardiac function. Under conditions of decreased blood flow, for instance through injury or atherosclerosis, blocking TSP1-CD47 interaction allows blood vessels to dilate and increases blood flow, tissue perfusion and tissue survival.11-22-2012
20110207795SMAD7 INHIBITOR COMPOSITIONS AND USES THEREOF - The present invention relates to the use of a specific inhibitor of Smad7 expression or function for the preparation of a pharmaceutical composition for the prevention, amelioration or treatment of a disease of the central nervous system and/or diseases related and/or caused by said disease of the central nervous system. Furthermore, methods for preventing, ameliorating and/or treating such diseases are disclosed.08-25-2011
20120295955RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF HER3 - The invention relates to oligomer compounds (oligomers), which target HER3 mRNA in a cell, leading to reduced expression of HER3 and/or HER2 and/or EGFR. Reduction of HER3 and/or HER2 and/or EGFR expression is beneficial for a range of medical disorders, such hyperproliferative disorders (e.g., cancer). The invention provides therapeutic compositions comprising oligomers and methods for modulating the expression of HER3 and/or HER2 and/or EGFR using said oligomers, including methods of treatment.11-22-2012
20120295958MODULATION OF GLUCAGON RECEPTOR EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of glucagon receptor. The compositions comprise oligonucleotides, targeted to nucleic acid encoding glucagon receptor. Methods of using these compounds for modulation of glucagon receptor expression and for diagnosis and treatment of disease associated with expression of glucagon receptor are provided.11-22-2012
20120295957PREVENTION OF TISSUE ISCHEMIA AND RELATED COMPOSITIONS - Provided herein are compositions for preventing, ameliorating, and/or reducing tissue ischemia and/or tissue damage due to ischemia, increasing blood vessel diameter, blood flow and tissue perfusion in the presence of vascular disease including peripheral vascular disease, atherosclerotic vascular disease, coronary artery disease, stroke and influencing other conditions, by suppressing CD47 and/or blocking TSP1 and/or CD47 activity or interaction. Influencing the interaction of CD47-TSP1 in blood vessels allows for control of blood vessel diameter and blood flow, and permits modification of blood pressure and cardiac function. Under conditions of decreased blood flow, for instance through injury or atherosclerosis, blocking TSP1-CD47 interaction allows blood vessels to dilate and increases blood flow, tissue perfusion and tissue survival.11-22-2012
20120295954TREATMENT OF INTERFERON REGULATORY FACTOR 8 (IRF8) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO IRF8 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Interferon Regulatory Factor 8 (IRF8), in particular, by targeting natural antisense polynucleotides of Interferon Regulatory Factor 8 (IRF8). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with The expression of IRF8.11-22-2012
20120295952TREATMENT OF FILAGGRIN (FLG) RELATED DISEASES BY MODULATION OF FLG EXPRESSION AND ACTIVITY - The present invention relates to antisense oligonucleotides and/or compounds that modulate the expression of and/or function of Filaggrin (FLG), in particular, by targeting natural antisense polynucleotides of Filaggrin (FLG). The invention also relates to the identification of these antisense oligonucleotides and/or compounds and their use in treating diseases and disorders associated with the expression of FLG.11-22-2012
20100144831BLOCKING OF GENE EXPRESSION IN EUKARYOTIC CELLS - The present invention provides methods for designing a sequence for efficient short interference RNA molecules. In particular, the present invention defines a universal target for siRNA derived from a poly A sequence, optionally in conjunction with unique sequences for gene silencing and inhibition of viral replication in a eukaryotic host cell. The present invention further provides methods for the treatment and prevention of diseases and disorders by silencing a gene of a virus, an oncogene, genes encoding transcription factors and many other diseases related genes. The present invention describes antisense nucleic acids compositions comprising sequences complementary to a target nucleic acid. The antisense sequences are designed to hybridize to complementary nucleic acid target regions in a target RNA, and inhibit translation, processing, transport, or binding by proteins or riboproteins. Target regions include, and are limited to a poly-A tail, and exclude, AUG, 5′ non-translated sequences, translation initiation factor binding sites, ribosome subunit binding sites, Shine Dalgarno sequence, 3′ nontranslated sequences, poly-addition site, 3′ cleavage site, coding region, intron, intron branch site, intron/exon junction, and splice sequence.06-10-2010
20090264506DELIVERY OF POLYNUCLEOTIDE AGENTS TO THE CENTRAL NERVOUS SYSTEM - The present invention provides a method for delivering polynucleotide agents, particularly oligonucleotides, to the CNS of a mammal by way of a neural pathway originating in the nasal cavity or through a neural pathway originating in an extranasal tissue that is innervated by the trigeminal nerve.10-22-2009
20090264505METHODS AND COMPOSITIONS FOR RNAI MEDIATED INHIBITION OF GENE EXPRESSION IN MAMMALS - Methods and compositions are provided for modulating, e.g., reducing, expression of a target sequence in mammals and mammalian cells. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is introduced into a target cell, e.g., by being administered to a mammal that includes the target cell, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.10-22-2009
20090264504RNA INTERFERENCE MEDIATED INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS (HIV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of human immunodeficiency virus (HIV) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HIV genes. The small nucleic acid molecules are useful in the treatment of HIV infection, AIDS, and/or diseases and conditions related to HIV infection and/or AIDS in a subject or organism.10-22-2009
20090264503SHORT INTERFERENCE RIBONUCLEIC ACIDS FOR TREATING ALLERGIC DISEASES - Provided herein are compounds, methods and compositions for treating allergic diseases. In particular, an isolated double stranded short interfering ribonucleic acid (siRNA) with a ribonucleotide sequence complementary to at least a portion of a target gene RNA such as an airway inflammation related-gene RNA, thereby resulting in the cleavage of the expressed target gene RNA via RNA interference mechanism, and is useful as a medicament for treating allergy by alleviating or minimizing airway inflammation of a subject.10-22-2009
20090264502COMPOSITIONS AND THEIR USES DIRECTED TO HSP27 - Disclosed herein are compounds, compositions and methods for modulating the expression of HSP27 in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.10-22-2009
20100137404Compositions and Methods for Altering Gene Expression - Compositions and methods for regulating gene expression in a growing animal are disclosed.06-03-2010
20100144840Methods for Modulating the Efficacy of Nucleic Acid Based Therapies - Covalently reactive antigen analogs are disclosed herein. The antigens of the invention may be used to stimulate production of catalytic antibodies specific for predetermined antigens associated with particular medical disorders. The antigen analogs may also be used to permanently inactivate endogenously produced catalytic antibodies produced in certain autoimmune diseases as well as in certain lymphoproliferative disorders. Also provided are methods for modulating the efficacy of nucleic acid based therapeutics.06-10-2010
20100144842RNA Interference Mediated Inhibition of NOGO and NOGO Receptor Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating NOGO and/or NOGO receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of NOGO and/or NOGO receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of NOGO and/or NOGO receptor genes, such as NOGO-A, NOGO-B, NOGO-C, NOGO-66 receptor, NI-35, NI-220, NI-250, myelin-associated glycoprotein, tenascin-R, and NG-206-10-2010
20120142756mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES - Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.06-07-2012
20110207797ENHANCED ANTISENSE OLIGONUCLEOTIDES - Described herein are gap-widened antisense oligonucleotides having improved therapeutic index as compared to 5-10-5 MOE gapmer antisense oligonucleotides of the same sequence. Also described are methods of reducing a target RNA in an animal using the gap-widened antisense oligonucleotides of the present invention. Further, are methods for selecting a gap-widened antisense oligonucleotides.08-25-2011
20120065248METHODS AND COMPOSITIONS INVOLVING MIRNA AND MIRNA INHIBITOR MOLECULES - The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.03-15-2012
20100144843RNAI THERAPEUTIC FOR RESPIRATORY VIRUS INFECTION - Double stranded siRNA molecules for combatting a respiratory virus, wherein the strands of an siRNA molecule may be from about 15 to about 60 nucleotides, and uses thereof. One strand of an siRNA molecule can be a nucleic acid sequence identical to a conserved site, or a variant thereof, within the nucleic acid sequence of the respiratory virus.06-10-2010
20090298914RNA Interference Mediated Inhibition of Severe Acute Respiratory Syndrome (SARS) Virus Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention comprises compounds, compositions, and methods useful for modulating the expression of genes associated with respiratory and pulmonary disease, such as severe acute respiratory syndrome (SARS) virus genes, using short interfering nucleic acid (siNA) molecules. This invention also comprises compounds, compositions, and methods useful for modulating the expression and activity of SARS virus genes, or other genes involved in pathways of SARS virus gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of SARS virus RNA.12-03-2009
20100081703TRI-DEMETHYLATION-CAPABLE PROTEIN COMPLEX, METHOD OF ITS PREPARATION AND ITS USE - The invention relates to a multiple-specificity demethylase complex comprising a Jumonji C (JMJC) domain-containing enzyme, a process of its preparation and its use.04-01-2010
20100004317METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.01-07-2010
20100022621CONSTRUCTION AND USE OF TRANSFECTION ENHANCER ELEMENTS - Nucleic acids comprising a nucleic acid moiety and two or more transfection enhancer elements (TEE's) according to the general formula (I): Hydrophobic moiety—pH-responsive hydrophilic moiety, wherein said pH sensitive hydrophilic moiety of said TEE is independently a weak acid having a pka of between 4 and 6.5 or is a zwitterionic structure comprising a combination of acidic groups with weak basis having a pKa of between 4.5 and 7.01-28-2010
20100004314USE OF SIRNA TO ACHIEVE DOWN REGULATION OF AN ENDOGENOUS GENE IN COMBINATION WITH THE USE OF A SENSE CONSTRUCT TO ACHIEVE EXPRESSION OF A POLYNUCLEOTIDE - The present invention relates to the combinatorial use of an siRNA targeted against an endogenous gene to knock out or knock down expression of the endogenous gene in a host and a delivery of a polynucleotide encoding the gene in a delivery vehicle/expression vector to the host to provide expression in the host of the protein encoded by the polynucleotide.01-07-2010
20100004316MULTIFUNCTIONAL CARRIERS FOR THE DELIVERY OF NUCLEIC ACIDS AND METHODS OF USE THEREOF - Described herein are multifunctional compounds useful as devices for the delivery of nucleic acids to cells. Also described herein are methods for using the multifunctional compounds.01-07-2010
20100004313Modified Poloxamers for Gene Expression and Associated Methods - Nucleotide delivery polymers, compositions, and associated methods for the enhancement of gene delivery and expression in solid tissues are provided. In one aspect, for example, a nucleotide delivery polymer may include a poloxamer backbone having a metal chelator covalently coupled to at least one terminal end of the poloxamer backbone. In another aspect, the nucleotide expression polymer has a metal chelator coupled to at least two terminal ends of the poloxamer backbone.01-07-2010
20090326044RNAi-Mediated Inhibition of Ocular Targets - RNA interference is provided for inhibition of ocular hypertension target mRNA expression for lowering elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. Ocular hypertension targets include carbonic anhydrase II, IV, and XII; β1- and β2 adrenergic receptors; acetylcholinesterase; Na12-31-2009
20090326040ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION - Methods for the rapid and long-term lowering of lipid levels in human subjects and for the treatment of conditions associated with elevated LDL-cholesterol and elevated apolipoprotein B are provided.12-31-2009
20090326045COMPOSITIONS AND METHODS FOR TOPICAL DELIVERY OF OLIGONUCLEOTIDES - The present invention relates to compositions and methods which enhance the delivery of oligonucleotides and other nucleosidic moieties via topical routes of administration. Preferred compositions include liposomes or penetration enhancers for the delivery of such moieties to dermal and/or epidermal tissue in an animal for investigative, therapeutic or prophylactic purposes.12-31-2009
20090326041COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF SGLT2 - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.12-31-2009
20110224282iRNA Agents Targeting VEGF - The features of the present invention relate to compounds, compositions and methods useful for modulating the expression of vascular endothelial growth factor (VEGF), such as by the mechanism of RNA interference (RNAi). The compounds and compositions include iRNA agents that can be unmodified or chemically-modified.09-15-2011
20110224278METHODS AND COMPOSITIONS FOR TREATING A SUBJECT FOR CENTRAL NERVOUS SYSTEM (CNS) INJURY - Methods for treating a central nervous system (CNS) injury in a subject are provided. Aspects of the methods include administering to the subject an effective amount of gamma aminobutyric acid (GABA) receptor signaling inhibitor to treat the subject for the CNS injury. Also provided are compositions finding use in embodiments of the methods. Methods and compositions of the invention find use in the treatment of a variety of different CNS injuries, including but not limited to, treating a subject for CNS injury associated with the occurrence of stroke.09-15-2011
20100210707Small Activating RNA Molecules and Methods of Use - The present invention provides compositions, pharmaceutical preparations, kits and methods for increasing expression of a gene product in a cell by contacting the cell with a small activating RNA (saRNA) molecule comprising a ribonucleic strand that is complementary to a non-coding nucleic acid sequence of the gene.08-19-2010
20110230544MODULATION OF C-REACTIVE PROTEIN EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of C-reactive protein. The compositions comprise oligonucleotides, targeted to nucleic acid encoding C-reactive protein. Methods of using these compounds for modulation of C-reactive protein expression and for diagnosis and treatment of disease associated with expression of C-reactive protein are provided.09-22-2011
20130217754PREVENTIVE OR THERAPEUTIC AGENT FOR FIBROSIS - Provided is siRNA effective for the treatment of fibrosis and a pharmaceutical containing the siRNA. An siRNA having a full length of 30 or fewer nucleotides and targeting a sequence consisting of 17 to 23 consecutive bases selected from the group consisting of bases at positions 1285 to 1318, bases at positions 1398 to 1418, bases at positions 1434 to 1463, bases at positions 1548 to 1579, bases at positions 1608 to 1628, bases at positions 1700 to 1726, bases at positions 1778 to 1798, bases at positions 1806 to 1826, and bases at positions 1887 to 1907 of SEQ ID NO: 1.08-22-2013
20130217752METHOD OF DERIVING MATURE HEPATOCYTES FROM HUMAN EMBRYONIC STEM CELLS - A method for producing mature hepatocytes having functional hepatic enzyme activity from human pluripotent cells is disclosed. The method includes the step of transferring an external vector comprising the DNA sequence coding for a microRNA having the seed sequence of the microRNA miR-122, the DNA sequence coding for a microRNA having the seed sequence of the microRNA miR-let-7c, a microRNA having the seed sequence of the microRNA miR-122, a microRNA having the seed sequence of the microRNA miR-let-7c, or a combination thereof into one or more fetal hepatocytes. The resulting cells differentiate into mature hepatocytes that exhibit functional hepatic enzyme activity, and can be used in drug metabolism and toxicity testing, in the study of viruses that target hepatic tissue, and as therapeutics.08-22-2013
20130217749MODULATION OF PHOSPHOENOLPYRUVATE CARBOXYKINASE-MITCHONDRIAL (PEPCK-M) EXPRESSION - Provided herein are methods, compounds, and compositions for reducing expression of phosphoenolpyruvate carboxykinase-mitochondrial (PEPCK-M) mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preventing or decreasing diabetes, obesity, metabolic syndrome, diabetic dyslipidemia, and/or hypertriglyceridemia in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of diabetes, obesity, metabolic syndrome, diabetic dyslipidemia, and/or hypertriglyceridemia, or a symptom thereof.08-22-2013
20090258926Methods and Compositions for Delivering siRNA into Mammalian Cells - Complex comprising a peptide carrier of SEQ ID NO:1 GALFLGFLGAAGSTMGAWSQPKR10-15-2009
20110144184PREVENTING OR TREATING VIRAL INFECTION USING AN INHIBITOR OF THE LSD1 PROTEIN, A MAO INHIBITOR OR AN INHIBITOR OF LSD1 AND A MAO INHIBITOR - An embodiment of the invention provides preventing or treating a viral infection of a host, comprising administering to the host an effective amount of an inhibitor of the protein LSD1 and/or a monoamine oxidase inhibitor. Another embodiment of the invention provides preventing or treating reactivation of a virus after latency in a host, comprising administering to the host an effective amount of an inhibitor of the protein LSD1 and/or a monoamine oxidase inhibitor. Another embodiment of the invention provides preventing or treating a viral infection in a mammal that has undergone, is undergoing, or will undergo an organ or tissue transplant, comprising administering to the mammal an effective amount of an inhibitor of the protein LSD1 and/or a monoamine oxidase inhibitor before, during, and/or after the organ or tissue transplant. The viral infection may be due to a herpesvirus, such as herpes simplex virus type 1 (HSV-1) or type 2 (HSV-2), varicella zoster virus (VZV), or cytomegalovirus (CMV). The viral infection may also be due to an adenovirus, including types 1-5.06-16-2011
20110224284PUTATIVE TUMOR SUPPRESSOR MICRORNA-101 MODULATES THE CANCER EPIGENOME BY REPRESSING THE POLYCOMB GROUP PROTEIN EZH2 - The present invention relates in general to microRNA profiling in disease. More specifically, the invention provides for methods and compositions of microRNA to inhibit the growth and formation of tumors.09-15-2011
20110224283ANTISENSE MODULATION OF NUCLEAR HORMONE RECEPTORS - Provided are antisense oligonucleotides and other agents that target and modulate nuclear hormone receptors (NHRs) such as the glucocorticoid receptor (GR), compositions that comprise the same, and methods of use thereof.09-15-2011
20110224279ANTICANCER AGENT - The present invention relates to a nucleic acid molecule and a pharmaceutical or diagnostic composition for the therapeutic and/or prophylactic treatment or diagnosis of cancer and/or metastasis thereof, comprising a nucleic acid molecule, or an amino acid sequence related to Trim71 and/or its mammalian and non mammalian orthologs and/or a nucleic acid sequence of the gene encoding for Trim71 and/or its mammalian and non mammalian orthologs.09-15-2011
20110230545EML4-ALK FUSION GENE - The present inventors found that a fusion gene present in some cancer patients is an oncogene. The present invention relates to a polypeptide as a novel fusion protein, a polynucleotide encoding the polypeptide, a vector comprising the polynucleotide, a transformed cell comprising the vector, a method for detecting the fusion protein or polynucleotide, a method for screening a therapeutic agent for cancer, and a method for treating cancer that is shown to be positive for the fusion gene. Further, the present invention relates kit, primer set, and probe useful in the detection of cancer that is shown to be positive for the fusion gene.09-22-2011
20110230543OLIGORIBONUCLEOTIDE INHIBITORS OF NRF2 AND METHODS OF USE THEREOF FOR TREATMENT OF CANCER - The invention provides novel double stranded oligoribonucleotides that inhibit the Nrf2 gene. The invention also provides a pharmaceutical composition comprising one or more such oligoribonucleotides, and a vector capable of expressing the oligoribonucleotide. The present invention also relates to methods and compositions for treating or preventing the incidence or severity of a cancerous disease, particularly various lung cancers.09-22-2011
20110230546dsRNA COMPOSITIONS AND METHODS FOR TREATING HPV INFECTION - The invention relates to a double-stranded ribonucleic acid (dsRNA) for treating human papilloma virus (HPV) infection. The dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of an HPV Target gene selected from among HPV E1, HPV E6 and the human E6AP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HPV infection and the expression of the E6AP gene using the pharmaceutical composition; and methods for inhibiting the expression of the HPV Target genes in a cell.09-22-2011
20090209627RNAi-MEDIATED INHIBITION OF CONNEXIN 43 FOR TREATMENT OF IOP-RELATED CONDITIONS - RNA interference is provided for inhibition of connexin 43 (Cx43) in intraocular pressure-related conditions, including ocular hypertension and glaucoma such as normal tension glaucoma and open angle glaucoma.08-20-2009
20090203765MODULATION OF EIF4E-BP2 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of eIF4E-BP2. The compositions comprise oligonucleotides, targeted to nucleic acid encoding eIF4E-BP2. Methods of using these compounds for modulation of eIF4E-BP2 expression and for diagnosis and treatment of diseases and conditions associated with expression of eIF4E-BP2 are provided.08-13-2009
20090221673Compositions and Methods for Regulating RNA Translation via CD154 CA-Dinucleotide Repeat - Compositions and methods for regulating CD154 gene expression are provided that rely on the interaction of hnRNP L with the CA-dinucleotide rich sequence of the 3′-untranslated region of CD154.09-03-2009
20090221677Methods for Reducing Body Fat and Increasing Lean Body Mass by Reducing Stearoyl-COA Desaturase 1 Activity - It is disclosed here that inhibiting the activity of the enzyme stearoyl-CoA desaturase (SCD1) in an animal causes the animal to have less body fat and greater lean body mass. The lower of SCD1 activity level can be accomplished by inhibiting activity of the enzyme or lowering levels of active enzyme in the subject.09-03-2009
20090203767INHIBITION STAT-1 - The present invention relates to inhibitors of the transcription factor STAT-1, their use as therapeutic means as well as their use for the prevention or therapy of cardio-vascular complications like restenosis after percutaneous angioplasty or stenosis of venous bypasses, the graft versus host reaction, the ischemia/refusion-related damage in the context of surgical interventions and organ transplantation respectively, immunological hypersensitivity reactions, in particular the allergic rhinitis, the drug and food allergies, in particular urticaria and celiac disease (sprue), contact eczema and the immune complex diseases, in particular alveolitis, arthritis, glomerulonephritis and allergic vasculitis, inflammatory chondro- and osteopathies, in particular arthrosis, gout, ostitis and osteomyelitis, polyneuritis as well as acute and subacute respectively, infection contingent and in particular post-infectious inflammatory diseases, in particular bronchitis, endocarditis, hepatitis, myocarditis, nephritis, pericarditis, peritonitis and pancreatitis, including the septic shock.08-13-2009
20120077867Compositions And Methods For Treating Pancreatic Cancer - The present invention provides a method of treating pancreatic cancer by inhibiting the activity cyclin D1 activity in tumor cells. The invention is based on the finding that cyclin D1 shRNA molecules are capable of attenuating tumor growth and interfering with tumor angiogenesis.03-29-2012
20120077866ANTISENSE MODULATION OF C-REACTIVE PROTEIN EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of C-reactive protein. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding C-reactive protein. Methods of using these compounds for modulation of C-reactive protein expression and for treatment of diseases associated with expression of C-reactive protein are provided.03-29-2012
20120077864RNAI-MEDIATED INHIBITION OF GREMLIN FOR TREATMENT OF IOP-RELATED CONDITIONS - RNA interference is provided for inhibition of gremlin in intraocular pressure-related conditions, including ocular hypertension and glaucoma such as normal tension glaucoma and open angle glaucoma.03-29-2012
20120077861Bispecific Antisense Oligonucleotides that Inhibit IGFBP-2 and IGFBP-5 and Methods of Using Same - Bispecific antisense oligonucleotides which consist essentially of a sequence of bases that is complementary to portions of both the gene encoding human IGFBP-2 and the gene encoding human IGFBP-5 are useful in as antisense therapeutics in the treatment of endocrine-regulated cancers.03-29-2012
20090209626Duplex Oligonucleotides with Enhanced Functionality in Gene Regulation - Disclosed are methods of enhancing functionality of duplex oligonucleotides and compositions made by the methods. The duplex oligonucleotides include siRNAs, miRNA mimics, and piRNA mimics which contain modified nucleotides and mismatches between the two strands of the molecule at specific nucleotide positions.08-20-2009
20090209624COMPOSITIONS AND THEIR USES FOR GENE THERAPY OF BONE CONDITIONS - In certain preferred embodiments, the present invention provides compositions and methods for the treatment of bone conditions associated with low bone density. In preferred embodiments, the present invention provides compositions and methods for the treatment of osteoprotegerin-responsive conditions.08-20-2009
20090239932Antisense oligonucleotides against protein kinase isoforms alpha, beta and gamma - Antisense compounds are provided for inhibiting PKB (protein kinase B) alpha, beta and gamma. The antisense compounds display high potency and selectivity. The antisense compounds do not suffer from problems of dimerisation or self-hybridization and have also been selected to not affect other enzymes. The antisense compounds may be used singularly or in combination to inhibit one, two or all of PKB alpha, beta and gamma and hence treat conditions in which these enzymes are important.09-24-2009
20090209623Anti-sense nucleic acid derived from organism - Disclosed are: a method for producing an anti-sense nucleic acid which has a wide variation in nucleotide sequences and excellent anti-sense properties at a low cost in a simple manner and in a large scale; and a cosmetic composition or pharmaceutical composition comprising the anti-sense nucleic acid. A method is discovered which can produce a low molecular weight nucleic acid having anti-sense properties from a nucleic acid starting material derived from an organism at a high yield by using a probe nucleic acid having at least a part of the nucleotide sequence of a target gene. An anti-sense nucleic acid produced by using a nucleic acid derived from an organism as a starting material; and a method for producing the anti-sense nucleic acid.08-20-2009
20090253775Compositions and Methods for Therapy and Diagnosis of Cancer and Cancer Metastasis - The present invention relates to methods which make possible to assess and/or prognose a cancer disease, the metastatic behaviour of a cancer disease and/or the occurrence of a relapse of cancer. In particular, the methods of the invention make possible to assess and/or prognose the occurrence of cancer metastasis, in particular distant metastasis. Preferably, the methods of the invention allow to discriminate malign from benign conditions.10-08-2009
20090221675USE OF IEX-1 FOR THE TREATMENT OF GLIOMA TUMORS - The present invention relates to a nucleic acid molecule encoding for IEX-1 polypeptide as a medicament. In a further aspect the present invention relates to a nucleic acid molecule encoding for IEX-1 polypeptide for the manufacture of a medicament for the treatment of gliomas.09-03-2009
20090253773RNA INTERFERENCE MEDIATED INHIBITION OF TNF AND TNF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating tumor necrosis factor and/or tumor necrosis factor receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of tumor necrosis factor and/or tumor necrosis factor receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of tumor necrosis factor and/or tumor necrosis factor receptor genes, (TNF and/or TNF receptor).10-08-2009
20090221676GLUCOSE-TRANSPORT RELATED GENES, POLYPEPTIDES, AND METHODS OF USE THEREOF - Methods and compositions for modulating glucose transport are provided herein.09-03-2009
20090221674COMPOSITIONS AND METHODS FOR THERAPY AND DIAGNOSIS OF CANCER - The present invention is directed to siRNA molecules which specifically target and cause RNAi-induced degradation of mRNA from TPTE genes, so that the protein product of the TPTE gene is not produced or is produced in reduced amounts. The siRNA compounds and compositions of the invention are useful for treating diseases which require inhibition of TPTE expression for their treatment, in particular cancer pathologies. The present invention also includes methods which make possible to assess and/or prognose the metastatic behaviour of a cancer disease and/or the occurrence of a relapse of cancer.09-03-2009
20090221672Biomarker for Prostate Cancer - The present invention provides a protein-based biomarker, Protein C Inhibitor (PCI) that is useful in qualifying prostate cancer status in a patient. In particular, the biomarker of this invention is useful to classify a subject sample as prostate cancer or non-prostate cancer. The biomarker can be detected by SELDI mass spectrometry.09-03-2009
20090247608Targeting Lipids - The present invention provides targeting lipids of structure10-01-2009
20090247609SM-PROTEIN BASED SECRETION ENGINEERING - The present invention concerns the field of cell culture technology. It describes a novel method for enhancing the secretory transport of proteins in eukaryotic cells by heterologous expression of Munc18c, Sly1 or other members of the SM protein family. This method is particularly useful for the generation of optimized host cell systems with enhanced production capacity for the expression and manufacture of recombinant protein products.10-01-2009
20090247607dsRNA COMPOSITIONS AND METHODS FOR TREATING HPV INFECTION - The invention relates to a double-stranded ribonucleic acid (dsRNA) for treating human papilloma virus (HPV) infection. The dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of an HPV Target gene selected from among HPV E1, HPV E6 and the human E6AP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HPV infection and the expression of the E6AP gene using the pharmaceutical composition; and methods for inhibiting the expression of the HPV Target genes in a cell.10-01-2009
20090239933HEPATITIS C ANTIVIRALS - The present invention relates to deoxyribozymes targeting and cleaving HCV RNA. More particularly, the present invention relates to deoxyribozymes and composition used for the inhibition of HCV replication and HCV-related diseases.09-24-2009
20090239934Anti-Myosin Va siRNA and Skin Depigmentation - The present invention relates to novel isolated siRNAs comprising a sense RNA strand and a complementary antisense RNA strand which together form an RNA duplex, characterized in that the sense RNA strand comprises a sequence which has at most one nucleotide that is distinct in relation to a fragment of 14 to 30 contiguous nucleotides of the nucleotide sequence of exon F of the gene encoding the myosin Va protein, and also to compositions comprising at least one such siRNA, and to the use of at least one such siRNA as a cosmetic or therapeutic agent for skin depigmentation.09-24-2009
20090253776siRNA targeting gremlin - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to CKSF1B1.10-08-2009
20090253770Target gene mimitin of myc - A new diagnosis or treatment of cancer is provided. The present invention provides a protein Mimitin which is a target of a cancer gene myc protein, a variant thereof and a fragment thereof, as well as a polynucleotide molecule encoding them. An inhibitory substance of biological activity of the present invention, which is afforded by the binding of a polynucleotide molecule encoding a Mimitin protein and Myc provides a useful means for the diagnosis, prophylaxis or treatment of cancer.10-08-2009
20090247610INTEGRASE COFACTOR - The present invention provides nucleic acid molecules which include a region specifically interacting with the nucleic acid encoding the LEDGF/P75 protein or the nucleic acid encoding a fragment of a LEDGF/P75 protein and methods and uses of such nucleic acid molecules.10-01-2009
20100273859TREATMENT AND PREVENTION OF HIV INFECTION - Disclosed herein are methods for treating and/or preventing HIV infection in a cell. The methods involve downmodulating one or more of the HIV-dependency factors (HDFs) disclosed herein to thereby treat and/or prevent HIV infection in the cell. Downmodulating the HDFs can be by contacting the cell with an agent that downmodulates the HDF. Also disclosed herein are methods for treating and/or preventing HIV infection in a subject comprising downmodulating one or more of the HIV-dependency factors (HDFs), disclosed herein, to thereby treat and/or prevent HIV infection in the subject. The method may further comprise selecting a subject diagnosed with or at risk for HIV infection, prior to downmodulating. Downmodulating the HDFs may comprise administering an agent that downmodulates the HDF to the subject such that the agent contacts HIV host cells of the subject. The agent may inhibit HDF gene expression, protein synthesis, HDF function or HDF activity, or combinations thereof.10-28-2010
20090258929Compositions and Methods for Modulating mTOR Signaling - The present invention relates to the use of antagonists of the mTOR signaling pathway and its constituent members (e.g., antagonists of MAPKAP) for the treatment, amelioration, and diagnosis of PI3K/AKT/mTOR-related disorders, e.g., cancers. The present invention also relates to methods of using modulators of mTOR (e.g., modulators of MAPKAP) for the treatment, amelioration, and diagnosis of PI3K/AKT/mTOR-related disorders, e.g., cancers. Assays for the identification of modulators of mTOR, MAPKAP, and hVPS34 are also provided.10-15-2009
20090258927Method for the Modulation of Function of Transcription Factors - There is provided a method of modulating the function of transcription factor by administering an effective amount of an oligonucleotide containing optimal nucleotide binding sites for the transcription factor. A therapeutic agent having an effective amount of an oligonucleotide for modulating function of transcription factors and a pharmaceutically acceptable carrier is also provided. Also provided is a treatment of patients having illnesses in which the activation of transcription factors play a role by administering to a patient an effective amount of an oligonucleotide which competitively binds the related transcription factor.10-15-2009
20100273857SUPPRESSION OF SCN9A GENE EXPRESSION AND/OR FUNCTION FOR THE TREATMENT OF PAIN - Disclosed herein are methods, sequences and nucleic acid molecules used to treat pain. Specifically, the methods and sequences include locally administering molecules that suppress the expression of amino acid sequences that encode for Na10-28-2010
20090258925NATURAL ANTISENSE AND NON-CODING RNA TRANSCRIPTS AS DRUG TARGETS - Small interfering RNA (siRNA) knock down antisense transcripts, and regulate the expression of their sense partners. This regulation can either be discordant (antisense knockdown results in sense transcript elevation) or concordant (antisense knockdown results in concomitant sense transcript reduction).10-15-2009
20090292002NUCLEOTIDE AND AMINO ACID SEQUENCES RELATING TO RESPIRATORY DISEASES AND OBESITY - This invention relates to genes identified from human chromosome 12q23-qter, which are associated with various diseases, including asthma. The invention also relates to the nucleotide sequences of these genes, isolated nucleic acids comprising these nucleotide sequences, and isolated polypeptides or peptides encoded thereby. The invention further relates to vectors and host cells comprising the disclosed nucleotide sequences, or fragments thereof, as well as antibodies that bind to the encoded polypeptides or peptides. Also related are ligands that modulate the activity of the disclosed genes or gene products. In addition, the invention relates to methods and compositions employing the disclosed nucleic acids, polypeptides or peptides, antibodies, and/or ligands for use in diagnostics and therapeutics for asthma and other diseases.11-26-2009
20100152279RNAi Inhibition of Serum Amyloid A For Treatment of Glaucoma - RNA interference is provided for inhibition of serum amyloid A mRNA expression in glaucomas involving SAA expression.06-17-2010
20100160412METHODS OF MODULATING METABOLIC MEMORY - Described are methods of identifying modulators of metabolic memory, for the treatment of microvascular complications of diabetes, as well as methods of use thereof. Also described are methods of treating microvascular complications of diabetes by decreasing expression and/or activity of SHP-1.06-24-2010
20120129914ORGANIC COMPOSITIONS TO TREAT HSF1-RELATED DISEASES - The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.05-24-2012
20100184828METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF RNA SILENCING - The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.07-22-2010
20100197765METHOD FOR PROMOTING THE EXPRESSION OF P53, AND P53 EXPRESSION PROMOTER FOR USE IN THE METHOD - A method for promoting expression of p53 mRNA and an expression promoting agent for use in the method are provided. The expression of the p53 gene can be promoted by inhibiting the binding between RNA transcribed from exon 1 of the vegf-A gene and mRNA transcribed from the p53 gene. The inhibition of the binding can be achieved by, for example, degrading the RNA transcribed from the exon 1 of the vegf-A gene, inhibiting transcription of RNA from the exon 1 of the vegf-A gene, or binding a nucleic acid that is different from the mRNA transcribed from the p53 gene to the RNA transcribed from the exon 1 of the vegf-A gene.08-05-2010
20100168209APOPTOSIS INDUCER FOR CANCER CELL - The present invention revealed that by suppressing the expression of the WRN gene, the BLM gene, or the RecQ1 gene, which belong to the RecQ helicase family, apoptosis is induced in various cancer cells and their proliferation is suppressed. Compounds that suppress the expression of RecQ helicase family genes or the functions of RecQ helicase proteins are thought to have the activity of inducing apoptosis.07-01-2010
20130190390MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD - Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.07-25-2013
20130190382METHODS FOR INHIBITING EXPRESSION OF CONNECTIVE TISSUE GROWTH FACTOR - This invention provides compounds which comprise modified oligonucleotides capable of inhibitory expression of connective tissue factor and composition containing same as well as methods of treating hyperprolific disorders and fibrotic diseases, and of reducing scarring resulting from wound healing using such compounds.07-25-2013
20100184829RNAI MODULATION OF SCAP AND THERAPEUTIC USES THEREOF - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a SCAP gene (Human SCAP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a SCAP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition; and methods for inhibiting the expression of a SCAP gene in a cell.07-22-2010
20100184822Method of modulating the activity of a nucleic acid molecule - The present invention relates, in general, to agents that modulate the pharmacological activity of nucleic acid molecules and, in particular, to agents that bind therapeutic or diagnostic nucleic acid molecules in a sequence independent manner and modulate (e.g., inhibit or reverse) their activity. The invention also relates to compositions comprising such agents and to methods of using same.07-22-2010
20110060031Compositions And Methods For Inhibiting Expression Of A Gene From The Ebola Virus - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the Ebola virus.03-10-2011
20110060030MODULATION OF APOLIPOPROTEIN C-III EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of apolipoprotein C-III. The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein C-III. Methods of using these compounds for modulation of apolipoprotein C-III expression and for diagnosis and treatment of disease associated with expression of apolipoprotein C-III are provided03-10-2011
20110060029METHOD OF TREATING CANCER BY MODULATING EPAC - Methods of treating cancer by preventing, mitigating, and/or inhibiting cancer metastasis in tumors expressing Epac by inhibiting the activity of exchange proteins directly activated by cyclic AMP (Epac) or one or more proteins within the Epac-induced carcinoma migration pathway.03-10-2011
20110060027Aptamer Therapeutics Useful in the Treatment of Complement-Related Disorders - The invention provides nucleic acid therapeutics and methods for using these nucleic acid therapeutics in the treatment of complement-related disorders.03-10-2011
20100240734METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.09-23-2010
20100197764ANTISENSE MODULATION OF ACYL COA CHOLESTEROL ACYLTRANSFERASE-2 EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of acyl CoA cholesterol acyltransferase-2. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding acyl CoA cholesterol acyltransferase-2. Methods of using these compounds for modulation of acyl CoA cholesterol acyltransferase-2 expression and for treatment of diseases associated with expression of acyl CoA cholesterol acyltransferase-2 are provided.08-05-2010
20100227912RNA INTERFERENCE MEDIATED INHIBITION OF MYOSTATIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating myostatin (GDF8) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of myostatin gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of myostatin genes.09-09-2010
20130123329MICRORNA COMPOSITIONS AND METHODS - Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of miR-34. Also provided herein are methods for the treatment of cancer.05-16-2013
20100204301Reducible polymers for nonviral gene delivery - Provided herein are biodegradable copolymers and nanoplex delivery systems comprising the same and a cargo molecule, such as a nucleic acid, a polynucleotide or other biomolecule. The biodegradable copolymers may comprise a reducible polymer linearly modified with lysine, such as a linear lysine-modified N,N′-cystamine bisacrylamide. The biodegradable copolymers also may be conjugated to a sequestering moiety, such as dietheylamine. The biodegradable copolymers also may comprise one or both of a targeting moiety and one or more moieties to facilitate endosomal escape. Also provided are methods for treating a pathophysiological condition and for increasing biocompatibility of a polymeric delivery system upon delivery to a subject using the biodegradable copolymers and nanoplexes.08-12-2010
20130123338NOVEL CATIONIC LIPIDS AND METHODS OF USE THEREOF - The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.05-16-2013
20130123344METHOD OF PREVENTING OR TREATING VIRAL INFECTION - Disclosed are compounds and pharmaceutical compositions containing compounds that inhibit JMJD2 proteins, including those of the formula (I):05-16-2013
20100222410Nuclease compositions and methods - The present invention generally relates to various nucleases and uses thereof, and in some cases, to the UPF0054 protein superfamily. Members of the UPF0054 protein superfamily, such as the 09-02-2010
20100216866RNAi Modulation of APOB and Uses Thereof - The invention relates to compositions and methods for modulating the expression of apolipoprotein B, and more particularly to the downregulation of apolipoprotein B by chemically modified oligonucleotides.08-26-2010
20100216865MicroRNA COMPOSITIONS IN THE TREATMENT OF VEGF-MEDIATED DISORDERS - The invention provides methods of treating diseases caused by the over-production of a VEGF polypeptide by administering miRNA or miRNA inhibitor compositions to decrease at least one activity of a VEGF polypeptide.08-26-2010
20100216867Methods of treatment of a bcl-2 disorder using bcl-2 antisense oligomers - The present invention is directed to the use of bcl-2 antisense oligomers to treat and prevent bcl-2 related disorders. These disorders include cancers, tumors, carcinomas and cell-proliferative related disorders. In one embodiment of the invention, a bcl-2 antisense oligomer is administered at high doses. The present invention is also directed to a method of preventing or treating a bcl-2 related disorder, in particular cancer, comprising administering a bcl-2 antisense oligomer for short periods of time. The present invention is further drawn to the use of bcl-2 antisense oligomers to increase the sensitivity of a subject to cancer therapeutics. The present invention also relates to pharmaceutical compositions comprising one or more bcl-2 antisense oligomers, which may comprise one or more cancer therapeutic agents.08-26-2010
20100216864RNA Antagonist Compounds for the Modulation of PCSK9 - The present invention provides compounds, compositions and methods for modulating the expression of PCSK9. In particular, this invention relates to oligomeric compounds, such as oligonucleotide compounds, which are hybridisable with target nucleic acids encoding PCSK9, and methods for the preparation of such oligomeric compounds. The oligonucleotide compounds have been shown to modulate the expression of PCSK9, and pharmaceutical preparations thereof and their use as treatment of hypercholesterolemia and related disorders are disclosed.08-26-2010
20100227908DIAGNOSTIC, PROGNOSTIC AND TREATMENT METHODS - The present invention relates generally to diagnostic and prognostic protocols for schizophrenia and its manifestations including sub-threshold phenotypes and states thereof. Profiling and stratifying individuals for schizophrenia and its various manifestations also form part of the present invention as well as monitoring and predicting efficacy of therapeutic, psychiatric, social or environmental intervention. The present invention further contemplates methods of treatment of schizophrenia and symptoms thereof.09-09-2010
20100227907Method of Treating Pain by Using Opioids and CAMKIV Inhibitors - A method of treating chronic pain in a mammal is provided comprising the steps of inhibiting CaMKIV in the mammal and administering to the mammal a therapeutically effective amount of an opioid.09-09-2010
20100227910GENE FUSION TARGETED THERAPY - The present invention relates to compositions and methods for cancer therapy, including but not limited to, targeted inhibition of cancer markers. In particular, the present invention relates to recurrent gene fusions as clinical targets for prostate cancer.09-09-2010
20100240733Histone Demethylation Mediated by the Nuclear Amine Oxidase Homolog LSD1 - LSD1, a homolog of nuclear amine oxidases, functions as a histone demethylase and transcriptional co-repressor. LSD1 specifically demethylates histone H3 lysine 4, which is linked to active transcription. Lysine demethylation occurs via an oxidation reaction that generates formaldehyde. Importantly, RNAi inhibition of LSD1 causes an increase in H3 lysine 4 methylation and concomitant de-repression of target genes, suggesting that LSD1 represses transcription via histone demethylation. The results thus identify a histone demethylase conserved from 09-23-2010
20100240731LIPOPEPTIDES FOR DELIVERY OF NUCLEIC ACIDS - Lipopeptide compounds comprising a peptide having 2 to 100 amino acid residues, and having a lipophilic group attached to at least one terminus of the peptide or to at least one amino acid residue of the peptide, and salts and uses thereof. The lipophilic group may be attached to the N-terminus, C-terminus or both termini of the peptide. The lipophilic group may be attached to at least one interal amino acid residue (i.e., an amino acid residue that is not the N-terminus or the C-terminus amino acid residue of the peptide). The lipophilic group may be attached to either termini or both and at least one internal amino acid residue.09-23-2010
20100222414SiRNA Sequence-Independent Modification Formats for Reducing Off-Target Phenotypic Effects in RNAi, and Stabilized Forms Thereof - Modification formats having modified nucleotides are provided for siRNA. Short interfering RNA having modification formats and modified nucleotides provided herein reduce off-target effects in RNA interference of endogenous genes. Further modification formatted siRNAs are demonstrated to be stabilized to nuclease-rich environments. Unexpectedly, increasing or maintaining strand bias, while necessary to maintain potency for endogenous RNA interference, is not sufficient for reducing off-target effects in cell biology assays.09-02-2010
20100222408Methods and Compositions for Treatment of Cystic Fibrosis - The present invention relates to compounds and methods of inhibiting p97/valosin-containing protein and compounds and methods of inhibiting gp78, the compounds and methods being useful for the treatment of a disorder comprising a IκB/NFκB mediated chronic inflammatory response component, for example cystic fibrosis.09-02-2010
20110112170OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOSIDES AND HAVING REDUCED TOXICITY - In certain embodiments, the present invention provides oligomeric compounds having favorable toxicity profiles and therapeutic indexes. Compounds of the present invention comprise bicyclic nucleosides. Certain such bicyclic nucleosides are pyrimidines that do not include a methyl group at the 5-carbon. Oligomeric compounds comprising such nucleosides are less toxic than compounds comprising bicyclic nucleosides that do include a methyl group at the 5-carbon. In certain embodiments, the present invention provides methods of preparing and using such compounds.05-12-2011
20100137406RNA Interference Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase (PDE4B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL-15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-1, MMP-2, MMP-3, MMP-9 and MMP-12. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), such as endogenous micro-RNA (miRNA) (e.g., miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC) including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.06-03-2010
20100144844RNAi-MEDIATED INHIBITION OF H1F1A FOR TREATMENT OF OCULAR ANGIOGENESIS - RNA interference is provided for inhibition of HIF1A mRNA expression for treating patients with ocular angiogenesis, particularly for treating retinal edema, diabetic retinopathy, sequela associated with retinal ischemia, posterior segment neovascularization (PSNV), and neovascular glaucoma, and for treating patients at risk of developing such conditions.06-10-2010
20100144836Methods for Detecting Epigenetic Modifications - A method of detecting a predisposition to, or the incidence of, cancer in a sample comprises detecting an epigenetic change in at least one gene selected from an NDRG4/NDRG2 subfamily gene, GATA4, OSMR, GATA5, SFRP1, ADAM23, JPH3, SFRP2, APC, MGMT, 11112, BNIP3, FOXE1, SYNE1, S0X17, PHACTR3 and JAM3, wherein detection of the epigenetic change is indicative of a predisposition to, or the incidence of, cancer. Also described are pharmacogenetic methods for determining suitable treatment regimens for cancer and methods for treating cancer patients, based around selection of the patients according to the methods of the invention. The present invention is also concerned with improved methods of collecting, processing and analyzing samples, in particular body fluid samples. These methods may be useful in diagnosing, staging or otherwise characterizing various diseases. The invention also relates to methods for identifying, diagnosing, staging or otherwise characterizing cancers, in particular gastrointestinal cancers such as colorectal cancers, gastric cancers and oesophageal cancers. The methods of the invention relate, inter alia, to isolating and analyzing the human DNA component from faecal samples and blood-based samples.06-10-2010
20100144833RNAi Modulation Of RSV, PIV And Other Respiratory Viruses And Uses Thereof - The present invention is based on the in vivo demonstration that RSV and PIV can be inhibited through intranasal administration of RNAi agents as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the present invention provides general and specific compositions and methods that are useful in reducing RSV or PIV mRNA levels, RSV or PIV protein levels and viral titers in a subject, e.g., a mammal, such as a human. These findings can be applied to other respiratory viruses.06-10-2010
20100144830CANCER CELL IDENTIFICATION MARKER AND CANCER CELL PROLIFERATION INHIBITOR - Disclosed is an identification marker which can be utilized for detection of various human cancer cells and whose expression closely relates to malignant alteration of cells, and compositions for human cancer treatment which are based on suppression of cancer cell proliferation through inhibition of expression of the identification marker. The marker is human heterochromatin protein 1γ (HP1γ), and the compositions for cancer treatment comprises one or more agents which suppresses the expression of human HP1γ gene, such as siRNAs to human HP1γ.06-10-2010
20090264501Methods and Compositions to Inhibit P2x7 Receptor Expression - Methods and compositions for the downregulation of P2X7 receptor expression or activity are disclosed. Preferred compositions comprise siNA. The methods and compositions are useful in the treatment of diseases characterised by increased 112X7 receptor activity, such as neuronal degeneration, Alzheimer's disease, inflammatory diseases, and some cancers.10-22-2009
20130217753AMPHIPHILIC MACROMOLECULES FOR NUCLEIC ACID DELIVERY - The invention provides amphiphilic macromolecules that are useful for delivering nucleic acids to cells and that are useful as delivery agents for gene therapy.08-22-2013
20130217756RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.08-22-2013
20120196920ANTISENSE OLIGONUCLEOTIDES AGAINST ACETYLCHOLINESTERASE FOR TREATING INFLAMMATORY DISEASES - The present invention relates to novel uses of antisense oligolucleotides targeted to the coding region of acetylcholinesterase (AChE) for treating inflammatory disorders other than inflammatory disorders of the central nervous system or the peripheral nervous system innervating voluntary muscles. More particularly, the present invention relates to uses of antisense oligodexoynucleotides targeted to AChE mRNA for treating inflammatory disease of the gastroinstestinal tract including inflammatory bowel disease.08-02-2012
20120196919EX-VIVO TREATMENT OF IMMUNOLOGICAL DISORDERS WITH PKC-THETA INHIBITORS - Disclosed is a method for treating a variety of diseases and disorders that are mediated or sustained through the activity of PKC-theta, including immunological disorders and atherosclerosis. Specifically, the invention relates to a method of treating an immunological disorder or atherosclerosis in a patient comprising treating blood from the patient, or a defined component of said blood, with an inhibitor of PKC-theta ex vivo and then re-administering the treated blood to the patient.08-02-2012
20110112168NOVEL SIRNA STRUCTURES - The invention relates to siRNA compounds possessing novel sequences and structural motifs which down-regulate the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.05-12-2011
20110112169RNAi-BASED THERAPEUTICS FOR ALLERGIC RHINITIS AND ASTHMA - The present invention provides compositions comprising one or more RNAi agents (e.g., siRNAs, shRNAs, or RNAi vectors) for the treatment of conditions and diseases mediated by (e.g., featuring IgE-mediated hypersensitivity), as well as systems for identifying RNAi agents effective for this purpose. The compositions are suitable for the treatment of allergic rhinitis and/or asthma. In certain embodiments of the invention the RNAi agent is targeted to a transcript that encodes a protein selected from the group consisting of the FCεRIα chain, the FCεRIβ chain, c-Kit, Lyn, Syk, ICOS, OX40L, CD40, CD80, CD86, Re1A, Re1B, 4-1BB ligand, TLR1, TLR2, TLR3, TLR4, TLR5, TLR6, TLR7, TLR8, TLR9, CD83, SLAM, common γ chain, and COX-2. In addition, the invention provides RNAi agent/delivery agent compositions and methods of use. In certain embodiments of the invention compositions comprising an RNAi agent are delivered by the respiratory route.05-12-2011
20110112171COMPOSITIONS AND THEIR USES DIRECTED TO PTPRU - Disclosed herein are compounds, compositions and methods for modulating the expression of PTPRU in a cell, tissue or animal. Also provided are methods of active target segment validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of diabetes, obesity, insulin resistance, insulin deficiency, hypercholesterolemia, hyperglycemia, hyperlipidemia, hypertriglyceridemia, hyperfattyacidemia, liver steatosis, steatohepatitis, non-alcoholic steatohepatitis, metabolic syndrome, cardiovascular disease and coronary heart diseaseby administration of antisense compounds targeted to PTPRU.05-12-2011
20120196918PREVENTING ISLET INFLAMMATION AND DYSFUNCTION AND MAINTAINING PROPER GLUCOSE LEVELS BY CONTROLLING eIF5A AND ITS HYPUSINATION - Pancreatic islet dysfunction, in both type 1 and type 2 diabetes results, in part, from cytokine-mediated inflammation leading to iNOS generation and the death of pancreatic islets. The production of pro-inflammatory cytokines involved in the generation of iNOS is facilitated by the availability of the hypusine-containing translational factor eIF5A, necessary for the maturation of antigen-presenting cells. Treatment with agents capable of interfering with the mRNA translating iNOS or with agents that can interfere with the hypusination of eIF5A, prevents the death of islets, lowers blood glucose levels, avoids insulin resistance, and generally avoids the inflammatory response in islets associated with type 1 and type 2 diabetes.08-02-2012
20100210711Methods and Compositions for the Treatment of Sterile Inflammation - Described are methods and compositions that inhibit IL-1 signalling for the treatment of acute inflammatory response to cell necrosis, and the attendant collateral tissue damage.08-19-2010
20100210710THERAPEUTIC siRNA MOLECULES FOR REDUCING VEGFR1 EXPRESSION IN VITRO AND IN VIVO - The invention relates to nucleic acid molecule compositions for use in modulating the expression and activity of VEGF pathway genes and decreasing unwanted neovascularization, including tumor angiogenesis, by RNA interference and methods and compositions comprising the nucleic acid molecules.08-19-2010
20100137408ANTISENSE ANTIBACTERIAL METHOD AND COMPOUND - A method and antisense compound for inhibiting the growth of pathogenic bacterial cells are disclosed. The compound contains no more than 12 nucleotide bases and has a targeting nucleic acid sequence of no fewer than 10 bases in length that is complementary to a target sequence containing or within 10 bases, in a downstream direction, of the translational start codon of a bacterial mRNA that encodes a bacterial protein essential for bacterial replication. The compound binds to a target mRNA with a T06-03-2010
20100137407SINGLE-CHAIN CIRCULAR RNA AND METHOD OF PRODUCING THE SAME - The present invention relates to a single-chain circular RNA having a sustained or slow-releasing RNA interference effect, characterized in that the single-chain circular RNA comprises a sense strand sequence, an antisense strand sequence complementary to the sense strand sequence, identical or different two loop sequences between the sense strand and the antisense strand, connecting both strands, wherein the sense strand and the antisense strand are paired to form a stem.06-03-2010
20130190383NUCLEIC ACID COMPOUNDS WITH CONFORMATIONALLY RESTRICTED MONOMERS AND USES THEREOF - This disclosure provides single-stranded and multi-stranded nucleic acid compounds having one or more double-stranded regions that regulate the function or expression of nucleic acid molecules expressed in a cell or a cell regulatory system dependent upon a nucleic acid. The disclosure provides a range of nucleic acid compounds having one or more conformationally restricted nucleomonomers (CRN). Certain nucleic acid compounds may have one or more conformationally restricted nucleomonomers and one or more hydroxymethyl substituted nucleomonomers (UNA). The nucleic acid compounds are useful in various therapeutic modalities.07-25-2013
20130190385METHOD FOR MODULATING DOUBLE-STRAND BREAK-INDUCED HOMOLOGOUS RECOMBINATION - The present invention concerns a method for modulating double-strand break-induced homologous recombination through the identification of effectors that modulate said double-strand break-induced homologous recombination by uses of interfering agents; these agents are capable of modulating double-strand break-induced homologous recombination through their respective actions on said effectors. The present invention also concerns the uses of these effectors and interfering agents and derivatives, respectively, by introducing them in an eukaryotic cell in order to modulate and more particularly to increase double-strand break-induced homologous recombination and gene targeting efficiency. The present invention also relates to specific derivatives of identified effectors and interfering agents, vectors encoding them, compositions and kits comprising such derivatives in order to modulate and more particularly to increase double-strand break-induced homologous recombination and gene targeting efficiency.07-25-2013
20130190381INHIBITORS OF LONG AND VERY LONG CHAIN FATTY ACID METABOLISM AS BROAD SPECTRUM ANTI-VIRALS - The present invention provides methods and compounds for treating viral infections using modulators of host cell enzymes relating to long chain fatty acid and lipid droplet metabolism. It includes a method of treating viral infections using triacsin C and its relatives, analogues and derivatives as well as other inhibitors of long chain fatty acid metabolism and lipid droplet metabolism.07-25-2013
20100222412MODULATION OF GLUCOCORTICOID RECEPTOR EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of glucocorticoid receptor. The compositions comprise antisense compounds, particularly antisense oligonucleotides which have particular in vivo properties, targeted to nucleic acids encoding glucocorticoid receptor. Methods of using these compounds for modulation of glucocorticoid receptor expression and for treatment of diseases are provided.09-02-2010
20100222407TRIBLOCK COPOLYMERS FOR CYTOPLASMIC DELIVERY OF GENE-BASED DRUGS - The invention features a triblock copolymer including a hydrophilic block; a hydrophobic block; and a positively charged block capable of reversibly complexing a negatively charged molecule, e.g., a nucleic acid, wherein the hydrophobic block is disposed between the hydrophilic block and the positively charged block. Desirably, the triblock copolymer is capable of self-assembling into a supramolecular structure, such as a micelle or vesicle. The invention further features methods of delivering negatively charged molecules and methods of treating a disease or condition using the polymers of the invention.09-02-2010
20100120894Intracellular DNA receptor - Provided herein are methods of identifying and using compounds that modulate an AIM2 polypeptide-mediated immune response. Further provided herein are methods of treating disease comprising administering to a patient a compound that decreases expression of an AIM2 polypeptide. Further provided herein are methods of providing gene therapy to a patient comprising administering to the patient a gene therapy agent and a compound that decreases expression of an AIM2 polypeptide. In certain embodiments, a compound that decreases expression of an AIM2 polypeptide comprises an siRNA or an shRNA.05-13-2010
20100063132SMALL INTERFERING RNA AND PHARMACEUTICAL COMPOSITION FOR TREATMENT OF HEPATITIS B COMPRISING THE SAME - The present invention relates to RNA interference mediated inhibition of Hepatitis B virus (HBV) by short interfering RNA (siRNA) molecules. Specially, siRNAs of the present invention which are double-stranded RNAs concern directing the sequence-specific degradation of viral RNA in mammalian cells. Disclosed is a DNA vector encoding the RNA molecules and synthesized siRNA molecules as well as method of therapeutic treatment for inhibition of HBV gene expression and viral replication by the administration of RNA molecules of the present invention.03-11-2010
20120142755COMPOSITIONS FOR PREVENTING, REDUCING OR TREATING KERATINOCYTE-MEDIATED INFLAMMATION - The present invention relates to the field of epidermal repair. More particularly, the invention concerns the use of a molecule able to inhibit a heteromeric receptor comprising OSMRβ as a subunit, for the preparation of a composition for inhibiting the expression of inflammatory factors by the keratinocytes. In particular, the invention concerns the use of antagonists and/or expression inhibitors of OSM, IL-17, TNFα, IL-31, IFN-γ, and/or the OSMRβ subunit, for the preparation of cosmetic or dermatologic compositions, especially for treating inflammatory skin diseases.06-07-2012
20120142757RNAi Modulation of AHA and Therapeutic Uses Thereof - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.06-07-2012
20120142753DIAGNOSIS AND TREATMENT OF ADRENOCORTICAL TUMORS USING HUMAN MICRORNA-483 - Disclosed herein are methods of diagnosing and treating a malignant adrenocortical tumor, including adrenocortical carcinoma. In some examples, methods of diagnosing a malignant adrenocortical tumor include detecting expression of at least one microRNA (miR) gene product, such as miR-100, miR-125b, miR-195, miR-483-3p, miR-483-5p and IGF2 mRNA in a sample obtained from the subject with an adrenocortical tumor and comparing expression of at least one of these miR gene products and IGF2 mRNA in the sample obtained from the subject to a control. Altered expression of at least one of the miR gene products and IGF2 mRNA, such as a decrease in miR-100, miR-125b or miR-195 or an increase in miR-483-3p, miR-483-5p, and an increase in IGF2 mRNA, in the sample obtained from the subject compared to the control indicates a malignant adrenocortical tumor.06-07-2012
20100144838Methods for Identifying Modulators of Pyrimidine Tract Binding Protein - Provided herein are methods and materials for identifying compounds that modulate polypyrimidine tract binding protein (PTB), a protein that functions as a negative regulator of pre-mRNA splicing by blocking the inclusion of numerous alternative exons into mRNA.06-10-2010
20110028531NOVEL SIRNA COMPOUNDS FOR INHIBITING RTP801 - The present invention provides chemically modified siRNA compounds that target RTP801 and pharmaceutical compositions comprising same useful for treating microvascular disorders, eye diseases, hearing impairment, neurodegenerative diseases and disorders, spinal cord injury and respiratory conditions.02-03-2011
20110028534RNAi INHIBITION OF CTGF FOR TREATMENT OF OCULAR DISORDERS - RNA interference is provided for inhibition of connective tissue growth factor mRNA expression in ocular disorders involving CTGF expression. Ocular disorders involving aberrant CTGF expression include glaucoma, macular degeneration, diabetic retinopathy, choroidal neovascularization, proliferative vitreoretinopathy and wound healing. Such disorders are treated by administering interfering RNAs of the present invention.02-03-2011
20110028535OLIGONUCLEOTIDES-TRANSFERRING PREPARATIONS - Preparations for transferring efficiently oligonucleotides necessary in antisense therapy or the like into animal cells so as to be useful in treatment for various diseases, which comprises a collagen as an essential component are provided.02-03-2011
20110028533NOVEL FER -LIKE PROTEIN, PHARMACEUTICAL COMPOSITIONS CONTAINING IT AND METHOD FOR ITS USE - Provided is a novel Fer-like protein, referred to as “FerC” (Fer colorectal cancer). FerC is a 47kDa protein having a unique N-terminal sequence and was found to be present in six colon cancer cell-lines and in five hepatocarcinoma (liver cancer) cell-lines, but not in CCD33 normal colon epithelial cells or normal human and mouse fibroblasts. Depletion of FerC impairs cell-cycle progression and induces apoptotic death in treated colon cancer (CC) cells. Also provided are nucleotide sequences that are antisense to at least a portion of the N-terminal sequence, short interfering nucleotides including such an antisense sequence, as well as pharmaceutical compositions containing an antisense sequence. The present pharmaceutical composition may be used in the treatment of cancer, in particular, colorectal cancer and liver cancer.02-03-2011
20100035965SIRNA INHIBITION OF P13K P85, PA110, AND AKT2 AND METHODS OF USE - The present invention provides polynucleotides, compositions including polynucleotides, and the uses thereof for treating cancer in a subject. The polynucleotides silence the expression of coding regions that encode polypeptides such as p85α, p110α, and Akt2. The cancers treatable using the methods described herein include colorectal cancer, breast cancer, lung cancer, and metastases thereof.02-11-2010
20090209620Direct Reversal Of The Suppressive Function Of CD4+Regulatory T Cells Via Toll-Like Receptor 8 Signaling08-20-2009
20090318538POLYMERIC OLIGONUCLEOTIDE PRODRUGS - Polymer conjugates containing nucleotides and/or oligonucleotides are disclosed.12-24-2009
20090318537SILENCING OF TGF BETA TYPE II RECEPTOR EXPRESSION BY SIRNA - The present application is directed to siRNA-based silencing of the type II receptor of TGFβ. siRNAs that target this receptor abrogate the receptor protein and transcript, TGFβ-mediated processes such as fibronectin assembly and cell migration also are inhibited and the molecules of the invention are efficacious in reducing the inflammatory response and matrix deposition. These findings show that siRNAs can be successfully delivered both in vitro and in vivo to regulate the TGFβ type II receptor level and modulate wound response. Methods and compositions exploiting the findings of the present invention have a wide-ranging application, extending from treatment of disorders of the eye to other organs and tissues throughout the body.12-24-2009
20090318532COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF PTP1B - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.12-24-2009
20090318536METHODS FOR TREATING HYPERCHOLESTEROLEMIA - Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.12-24-2009
20110130440NON-NATURAL RIBONUCLEOTIDES, AND METHODS OF USE THEREOF - One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such modified nucleosides. Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.06-02-2011
20120129915ORGANIC COMPOSITIONS TO TREAT HSF1-RELATED DISEASES - The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.05-24-2012
20100222409COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF PRO-APOPTOTIC GENES - The invention relates to one or more inhibitors, in particular siRNA compounds, which down-regulate the expression of a pro-apoptotic gene selected from the group consisting of TP53; HTRA2; KEAP1; SHC1-SHC, ZNHIT1, LGALS3 and HI95. The invention also relates to a pharmaceutical composition comprising the compound, and a pharmaceutically acceptable carrier. The present invention further provides methods of treating a subject afflicted with a disease or a condition associated with those genes, comprising administering to the subject a pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.09-02-2010
20130131139ROR1 AS A GENE TARGET IN ACUTE LYMPHOBLASTIC LEUKEMIA - Disclosed are methods of selecting a subject suspected of having or having leukemia, such as lymphoblast leukemia (B-ALL), for treatment with an agent that inhibits ROR1-regulated signaling activity. In some examples, cells obtained from the subject are screened for over expression of ROR1. In other examples, the cells are contacted with an agent that inhibits ROR1 signaling activity and a ROR1-regulated signaling activity is detected. An alteration in the ROR1-regulated signaling activity as compared to a control identifies the subject as one that would benefit from treatment with an agent that inhibits ROR1 signaling activity. Also disclosed are methods for identifying an agent for treating a subject with a ROR1-dependent leukemia or with a predisposition for developing a ROR1-dependent leukemia, and methods for treating or inhibiting a ROR1-dependent leukemia, such as B-ALL in a subject.05-23-2013
20130131140TREATMENT OF DIABETES AND DISORDERS ASSOCIATED WITH VISCERAL OBESITY WITH INHIBITORS OF HUMAN ARACHIDONATE 12 LIPOXYGENASE AND ARACHIDONATE 15-LIPOXYGENASE - A basis for understanding the arachidonate 12-lipoxygenase pathway, as well as and methods and kits for inhibiting the arachidonate 12-lipoxygenase pathway for the treatment, reversal, reduction, modulation or prevention of disease states and conditions related to type 1 or type 2 diabetes, are disclosed. Also disclosed are inflammatory forms of ALOX12 and 15, which are selectively expressed in omental adipose tissue of obese humans. Inhibitors of ALOX12 and 15 can be used to treat, prevent, modulate or reduce complications associated with increased visceral obesity and inflammation, including type 2 diabetes. Also disclosed are methods for developing selective ALOX inhibitors for treating or reducing complications associated with increased visceral obesity and inflammation.05-23-2013
20130131142RNA INTERFERENCE IN OCULAR INDICATIONS - The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.05-23-2013
20130131143RTP801L SIRNA COMPOUNDS AND METHODS OF USE THEREOF - The invention provides chemically modified siRNA oligonucleotides that target RTP801L, compositions comprising same and to the use of such molecules to treat, inter alia, respiratory diseases including acute and chronic pulmonary disorders, eye diseases including glaucoma and ION, microvascular disorders, angiogenesis- and apoptosis-related conditions, and hearing impairments.05-23-2013
20130131144GDE Compositions and Methods - The present invention relates to compositions to treat glycerophosphodiester phosphodiesterase (GDE) related disorders. The invention also relates to methods treating GDE related disorders. The invention further relates to kits for treating GDE related disorders in a subject. The invention further relates to methods of identifying novel treatments for treating GDE related disorders in a subject.05-23-2013
20130131145COMPOSITIONS AND METHODS FOR ALTERING RNAi - The present invention relates to compositions and methods for altering (e.g., enhancing) RNAi. In particular, the present invention provides systems and methods for identifying regulators of RNAi. For example, the present invention provides RNAi regulators (e.g., HPS1 and HPS4) and methods of altering (e.g., inhibiting) these regulators in order to alter (e.g., enhance) RNAi. The present invention also provides methods of identifying inhibitors (e.g., small molecule, nucleic acid (e.g., siRNA), and antibody) of RNAi regulators and methods of using the same (e.g., to enhance RNAi). Compositions and methods of the present invention find use in research (e.g., functional genomics), therapeutic (e.g., drug discovery and delivery) and clinical applications.05-23-2013
20130131146Compositions and Methods for Treating Pulmonary Conditions - The present invention relates to the role of cystic fibrosis transmembrane conductance regulator (CFTR) in pulmonary conditions. In one embodiment, a method for assessing the severity of lung damage from a pulmonary condition in a subject comprises the steps of (a) measuring the level and/or functional activity of membrane/lipid-raft cystic fibrosis transmembrane conductance regulator (CFTR)in a sample from the subject; (b) measuring the level of ceramide or its species in a sample from the subject; and (c) comparing the membrane/lipid- raft CFTR level and/or functional activity and ceramide level to a control sample, wherein a difference in membrane/lipid-raft CFTR level and/or functional activity and ceramide level is indicative of the severity of lung damage. The method can further comprise treating the subject based on the severity of lung damage. In particular embodiments, the treatment comprises administering a CFTR agonist and/or an agent that inhibits the synthesis of ccramide or its species.05-23-2013
20130131148MICRO-RNA FOR CANCER DIAGNOSIS, PROGNOSIS AND THERAPY - The present invention relates to polymorphic binding sites for micro-RNA and to micro-RNA-based cancer diagnosis, cancer therapy and personalized medicine. In particular, the present invention relates to diagnosis, therapy and personalized medicine with the specific miR-515-5p molecule.05-23-2013
20130131149METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF ANDROGEN RECEPTOR BY DOUBLE-STRANDED RNA - This invention relates to compounds, compositions, and methods useful for reducing AR target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.05-23-2013
20100298406Method of suppressing tumour growth - A method of suppressing undesirable cell proliferation, such as tumour growth, is provided comprising the step of increasing the level of PCL2 in target cells.11-25-2010
20100311812RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of ICAM gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of ICAM genes.12-09-2010
20110009467Methods to Increase or Decrease Bone Density - The SOST gene gives rise to sclerostin, a protein that leads to apoptosis of bone progenitor cells. The invention provides antagonists to the sclerostin protein, and methods for identifying new sclerostin antagonists. The invention also provides molecules that can depress expression of the SOST gene, as well as methods for identifying such molecules. Such molecules and antagonists are useful for increasing bone mineralization in mammals, for example, in the treatment of osteoporosis.01-13-2011
20100324117HEPATITIS C DSRNA EFFECTOR MOLECULES, EXPRESSION CONSTRUCTS, COMPOSITIONS, AND METHODS OF USE - The present invention provides agents, compositions, constructs and methods for silencing HCV polynucleotides, as well as methods and compositions for treating or preventing HCV infection in a mammalian cell. In one aspect, the present invention provides an agent or composition comprising at least one double-stranded RNA effector molecule or complex. The double-stranded RNA effector molecule or complex comprises: (1) a sequence of at least 19 nucleotides having at least 90% identity with a nucleotide sequence within HCV Conserved Region 1 (SEQ ID NO: 2), HCV Conserved Region 2 (SEQ ID NO: 3), HCV Conserved Region 5 (SEQ ID NO: 4), (ATR)-1 (SEQ ID NO: 86), ATR-2 (SEQ ID NO: 87), ATR-3 (SEQ ID NO: 88), ATR-4 (SEQ ID NO: 89); and (2) its complementary sequence. In another aspect, the present invention provides a construct suitable for replication in a host cell, and/or suitable for expression of an RNA molecule or complex of the invention in vitro or in vivo. In a third aspect, the present invention provides a method for silencing HCV RNA in a mammalian cell, which comprises administering to the mammalian cell an agent, composition, or construct of the invention in a manner and amount effective to silence HCV RNA in the cell. In a related aspect, the invention provides a method for treating or preventing HCV infection in a patient, comprising administering to the patient an effective amount of an agent, composition, or construct of the invention as described herein.12-23-2010
20130137749Compositions and Methods for Gene Silencing - Compositions and methods for modulating the expression of a protein of interest are provided.05-30-2013
20130137751TREATMENT OF GLIAL CELL DERIVED NEUROTROPHIC FACTOR (GDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO GDNF - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Glial cell derived neurotrophic factor (GDNF), in particular, by targeting natural antisense polynucleotides of Glial cell derived neurotrophic factor (GDNF). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of GDNF.05-30-2013
20130137752RNA Interference Mediated Inhibition of Catenin (Cadherin-Associated Protein), Beta 1 (CTNNB1) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of CTNNB1 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against CTNNB1 gene expression.05-30-2013
20130137753METHODS AND COMPOSITIONS FOR THE DIAGNOSIS AND TREATMENT OF BREAST CANCER - Embodiments of the present disclosure relate to methods and compositions for the diagnosis and treatment of breast cancer. In some embodiments, the present disclosure relates to the use of Merlin, OPN and particular microRNAs for evaluating the presence of breast cancer in a subject and for identifying therapeutic compounds.05-30-2013
20100311811RNA-MEDIATED EPIGENETIC REGULATION OF GENE TRANSCRIPTION - The invention provides a method of regulating transcription of a gene that is a target for an epigenetic regulator; a method of characterizing the transcriptional activity of such a gene; a method of screening for a chromosomal element (CE) for an epigenetic regulator of a target gene; an isolated complex including an epigenetic regulator for a target gene, wherein the epigenetic regulator is specifically bound to a non-coding polynucleotide; and a method of screening for a modulator of transcription of a gene that is a target for an epigenetic regulator.12-09-2010
20100311807METHODS FOR IDENTIFYING DIABETES AND OBESITY THERAPEUTICS - The invention relates to inhibition of Par-1b kinase activity for treating disorders including diabetes and obesity. The invention also relates to screening for compounds or compositions that inhibit the kinase activity of Par-1b protein, which compounds and compositions are useful in the treatment of diabetes and obesity, as well as preparing compounds for treatment of diabetes and obesity.12-09-2010
20130143944CHEMO- AND RADIATION-SENSITIZATION OF CANCER BY ANTISENSE TRPM-2 OLIGODEOXYNUCLEOTIDES - Administration of antisense oligodeoxynucleotides (ODN) targeted against the testosterone-repressed prostate message-2 (TRPM-2) gene can reduce the amount of TRPM-2 in renal cell cancer (RCC) cells and other cancer cells, and as a result enhance chemosensitivity of these cells to chemotherapy agents and radiation. Thus, for example, the sensitivity of renal cell cancer cells to a chemotherapeutic agent can be increased by exposing renal cell cancer cells to a chemotherapeutic agent and an agent which reduces the amount of TRPM-2 in the renal cell cancer cells. This provides an improved method for treatment of renal cell cancer, which is generally resistant to treatment with known chemotherapy agents.06-06-2013
20130143946TREATMENT OF DISCS LARGE HOMOLOG (DLG) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO DLG - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Discs large homolog (DLG), in particular, by targeting natural antisense polynucleotides of Discs large homolog (DLG). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of DLG.06-06-2013
20130143947Injectable Pharmaceutical Composition for Preventing, Stabilising and/or Inhibiting Pathological Neovascularization-Related Conditions - This invention relates to a pharmaceutical composition for the treatment and/or prevention of at least one pathological neovascularization-related conditions of the interior of the eye, the composition comprising a therapeutically effective amount of an antisens oligonucleotide having the sequence SEQ ID NO: 1: 5′-TCTCCGGAGGGCTCGCCATGCTGCT-3′ or any function conservative sequence comprising from 9 to 30 nucleotides that has 75%, 80%, 85%, 90%, 95% or more than 95%, 96%, 97%, 98%, 99% of identity compared to SEQ ID NO: 1 and that conserves the capacity of inhibiting IRS-1 gene expression as SEQ ID NO: 1, and the composition being administered within the posterior segment of the eye to a subject in need thereof; this invention also relates to a method for treating a pathological neovascularization-related condition of the interior of the eye in a subject in need thereof comprising administering to the subject a therapeutically effective amount of said pharmaceutical composition.06-06-2013
20130143948MST1 AS A PROGNOSTIC BIOMARKER AND THERAPEUTIC TARGET IN HUMAN CANCER - The present invention relates to MST1 and MST2 cancer biomarkers. The inventors demonstrate herein that MST1 and/or MST2 can be used as biomarkers for the detection and prognosis of prostate cancer. The invention further discloses that enforced expression of MST1 can be used to inhibit and/or suppress the progression of prostate cancer.06-06-2013
20100324121METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene.12-23-2010
20110213006Compositions and Methods for Treatment of Uncontrolled Cell Growth - Compositions and methods are provided for the treatment of cancer and other diseases of uncontrolled cell growth. Inhibitors of t-RNA and 28s rRNA are provided as are non-functional amino acid residues for charging of t-RNA molecules. Therapeutic application of the above inhibitors is also provided for the treatment of cancer.09-01-2011
20110003882RNAi Modulation of AHA and Therapeutic Uses Thereof - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.01-06-2011
20100331394Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.12-30-2010
20100331391ZAP-70 AS PREDICTOR AND MODULATOR OF EFFECTOR FUNCTION OF T CELLS - In this application is described a novel, multiparameter analysis of TCR-coupled signaling and function in resting and activated naive and memory CD4 T cells, revealing a biochemical basis for immunological recall. Results reveal a novel biochemical signature imparted to memory CD4 T cells enabling efficacious responses through increased ZAP-70 expression and reduced accumulation of downstream signaling events.12-30-2010
20100331389Compositions and methods for the specific inhibition of gene expression by dsRNA containing modified nucleotides - The invention features compositions and methods that are useful for reducing the expression or activity of a specified gene in a eukaryotic cell.12-30-2010
20110009472RNAi Probes Targeting Cancer-Related Proteins - RNAi sequences that are useful as therapeutics in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma and melanoma; and Alzheimer's disease. These sequences target clusterin, IGFBP-5, IGFBP-2, both IGFBP-2 and -5 simultaneously, Mitf, and B-raf. The invention further provides for the use of these RNAi sequences in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma and melanoma; and Alzheimer's disease, and a method of treating such conditions through the administration of the RNA molecules with RNAi activity to an individual, including a human individual in need of such treatment.01-13-2011
20110245318RNA SEQUENCE-SPECIFIC MEDIATORS OF RNA INTERFERENCE - The present invention relates to a 10-06-2011
20100184827METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF RNA SILENCING - The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.07-22-2010
20100184825RNA Interference Mediated Inhibition of Protein Tyrosine Phosphatase-1B (PTP-1B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating protein tyrosine phosphatase-1B (PTP-1B) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of PTP-1B gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of PTP-1B genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing obesity, insulin resistance, diabetes (eg. type II and type I diabetes) in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of PTP-1B in a cell or tissue, alone or in combination with other treatments or therapies.07-22-2010
20100184821Compositions and Methods for Modulating Angiogenesis - The invention generally features compositions and methods that are useful for modulating angiogenesis.07-22-2010
20100184820COMBINATIONS COMPRISING STAUROSPORINES - The present invention relates to a method of treating myelodysplastic syndromes, lymphomas and leukemias, and also solid tumors with a pharmaceutical combination of a FLT-3 kinase inhibitor and an antisense oligonucleotide or a mcl-1-specific RNAi construct. It also relates to the use of a pharmaceutical combination of a histone deacetylase inhibitor and a FLT-3 kinase inhibitor for the treatment of the diseases or malignancies mentioned above and the use of such a pharmaceutical composition for the manufacture of a medicament for the treatment of these diseases or malignancies.07-22-2010
20110245325PHARMACEUTICAL COMPOSITION FOR TREATMENT OF CANCER AND ASTHMA - Provided is a pharmaceutical composition for treating cancer and asthma.10-06-2011
20110245323RIG-Like Helicase Innate Immunity Inhibits VEGF-Induced Tissue Responses - The present invention encompasses compositions and compounds as well as methods of their use for the regulation of a VEGF-induced tissue response. A VEGF-induced tissue response may include angiogenesis, inflammation, increased vascular permeability, increased vascular leak, hemorrhage, or mucus metaplasia. As such, the present invention encompasses methods of treating diseases where a VEGF-induced tissue response is part of the disease's clinical presentation. Specifically, the present invention provides compounds and compositions as well as methods for treating acute lung injury (ALI), acute respiratory distress syndrome (ARDS), asthma, chronic obstructive pulmonary disease (COPD), obstructive sleep apnea (OSA), idiopathic pulmonary fibrosis (IPF), tuberculosis, pulmonary hypertension, pleural effusion, and lung cancer.10-06-2011
20110245322METHODS FOR IDENTIFYING AND COMPOUNDS USEFUL FOR INCREASING THE FUNCTIONAL ACTIVITY AND CELL SURFACE EXPRESSION OF CF-ASSOCIATED MUTANT CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR - The present invention relates to agents, and methods for identifying compounds, which agents and compounds result in the modulation of cellular trafficking of proteins in particular that of CF-associated mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). In addition, the invention relates to compositions and methods for the use thereof in treating conditions that are characterized by an ER-associated protein misfolding and abnormal cellular trafficking of disease-associated proteins, including cystic fibrosis (CF).10-06-2011
20110245321SIDEROPHORE-MEDIATED IRON UPTAKE IN BACTERIAL INFECTION - The present invention relates to methods of inhibiting 10-06-2011
20110245320NUCLEASE RESISTANT DOUBLE-STRANDED RIBONUCLEIC ACID - This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.10-06-2011
20110245319RNAi-Mediated Inhibition of RHO Kinase for Treatment of Ocular Disorders - RNA interference is provided for inhibition of Rho kinase mRNA expression for treating patients with ocular disorders, particularly for treating intraocular pressure, ocular hypertension and glaucoma. Rho kinase mRNA targets include mRNA for ROCK1 and ROCK2.10-06-2011
20110009470Nucleic acid external skin formulation - The present invention provides a nucleic acid external skin formulation having a high skin permeability and which is prepared from a polymeric nucleic acid having a high molecular weight and sodium alginate. The nucleic acid external skin formulation is highly skin permeable and delivers its active ingredient nucleic acid to the affected area efficiently. The nucleic acid used may be smaller in amount, and thus, the formulation is also superior in safety and cost. The working mechanism thereof is different from that of the low-molecular weight compounds currently used as an active ingredient for skin anti-inflammatory agents, and thus, the formulation may be applicable to cases where such low-molecular weight medicines are less effective.01-13-2011
20110034533COMPOSITIONS AND METHODS MODULATING MG29 FOR THE TREATMENT OF DIABETES - Disclosed herein are compositions and methods for treatment of muscle dysfunction, including diabetes. In addition, the invention relates to therapeutic compositions comprising nucleotides and/or polypeptides of the invention in combination with a pharmaceutically acceptable carrier, wherein the composition facilitates the treatment of skeletal muscle disorders. Moreover, the invention relates to the treatment and/or prevention of pathological conditions associated with altered intracellular Ca2+ regulation and disrupted membrane structure that occurs when the expression levels of MG29 are reduced.02-10-2011
20110034535POSITIVE CONTROLS FOR EXPRESSION MODULATING EXPERIMENTS - The invention pertains to the use of an apoptosis inducing combination of at least a. a first expression modulating compound silencing the expression of at least a first target gene involved in apoptosis and b. a second expression modulating compound silencing the expression of at least a second target gene involved in apoptosis as a positive control in expression modulating assays. Also provided are suitable methods, kits and compositions.02-10-2011
20110034537COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF CD45 GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the CD45 gene.02-10-2011
20110034534siRNA compounds and methods of use thereof - The present invention relates to compounds, pharmaceutical compositions comprising same and methods of use thereof for the inhibition of certain genes, including SOX9, ASPP1, CTSD, CAPNS1, FAS and FAS ligand. The compounds and compositions are useful in the treatment of subjects suffering from diseases or conditions and or symptoms associated with diseases or conditions in which gene expression has adverse consequences.02-10-2011
20110034536CONTROLLING THE POTENTIAL OF PRIMATE NEURAL STEM CELLS BY REGULATING PAX6 - A transcription factor both necessary and sufficient for human neuroectoderm specification, Pax6, as well as applications thereof, is disclosed.02-10-2011
20110034538MicroRNA-Based Methods and Compositions for the Diagnosis, Prognosis and Treatment of Gastric Cancer - Methods and compositions for the diagnosis, prognosis and/or treatment of gastric cancer associated diseases are disclosed.02-10-2011
20110034532Modulation of T Cell Signaling Threshold and T Cell Sensitivity to Antigens - MicroRNAs (miRNAs) are a diverse and abundant class of ˜22-nucleotide (nt) endogenous regulatory RNAs that play a variety of roles in animal cells by controlling gene expression at the posttranscriptional level. Increased miR-181a expression in mature T cells is shown to cause a marked increase in T cell activation and augments T cell sensitivity to peptide antigens. Moreover, T cell blasts with higher miR-181a expression become reactive to antagonists. The effects of miR-181a on antigen discrimination are in part achieved by dampening the expression of multiple negative regulators in the T cell receptor (TCR) signaling pathway, including PTPN22 and the dual specificity phosphatases DUSP5 and DUSP6. This results in a reduction in the TCR signaling threshold, thus quantitatively and qualitatively enhancing T cell sensitivity to antigens.02-10-2011
20110039915IMMUNOSTIMULATORY siRNA MOLECULES - The present invention relates to a double-stranded siRNA molecule that is capable of silencing gene expression as well as inducing an immune response. The molecule comprises a sense strand and an antisense strand, wherein the antisense strand comprises a first nucleotide sequence that is specifically complementary to mRNA transcribed from a target gene, and the sense strand comprises a second nucleotide sequence that is substantially or perfectly complementary to the antisense strand with the exception that the second nucleotide sequence comprises at least one immunostimulatory motif comprising two or more non-complementary nucleotides. Such a molecule may be utilized in a method of treating or preventing a disease or condition (such as a viral infection, a bacterial infection, or cancer) in a subject.02-17-2011
20110039914MODIFIED RNAI POLYNUCLEOTIDES AND USES THEREOF - The invention relates to improved RNAi constructs and uses thereof. The construct has a double stranded region of 19-49 nucleotides, preferably 25, 26, or 27 nucleotides, and preferably blunt-ended. The construct has selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. For example, the sense strand may be modified (e.g., one or both ends of the sense strand is/are modified by four 2′-O-methyl groups), such that the construct is not cleaved by Dicer or other RNAse III, and the entire length of the antisense strand is loaded into RISC. In addition, the antisense strand may also be modified by 2′-O-methyl group at the 2nd 5′-end nucleotide to greatly reduce off-target silencing. The constructs of the invention largely avoids the interferon response and sequence-independent apoptosis in mammalian cells, exhibits better serum stability, and enhanced target specificity.02-17-2011
20110039913ANTISENSE MODULATION OF HYDROXYSTEROID 11-BETA DEHYDROGENASE 1 EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of hydroxysteroid 11-beta dehydrogenase 1. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding hydroxysteroid 11-beta dehydrogenase 1. Methods of using these compounds for modulation of hydroxysteroid 11-beta dehydrogenase 1 expression and for treatment of diseases associated with expression of hydroxysteroid 11-beta dehydrogenase 1 are provided.02-17-2011
20110039910MODULATION OF APOLIPOPROTEIN (A) EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of apolipoprotein(a). The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein(a). Methods of using these compounds for modulation of apolipoprotein(a) expression and for diagnosis and treatment of disease associated with expression of apolipoprotein(a) are provided.02-17-2011
20110039909METHODS AND MATERIALS FOR REDUCING GLI2 EXPRESSION - Methods and materials for reducing expression of GLI2 are disclosed including nucleic acid molecules such as short hairpin RNAs that direct cleavage of GLI2 encoding transcripts and the use of such molecules for reducing prostate cancer cell growth.02-17-2011
20110039911METHOD OF INHIBITING NONSENSE-MEDIATED mRNA DECAY - Provided are methods for treating a NAD comprising administering to a patient suffering from a NAD a composition comprising an eIF5A inhibitor compound in an amount effective to prevent intracellular hypusination of eIF5A, whereby gene expression of NMD-susceptible mRNA is increased.02-17-2011
20130190387TREATMENT OR PREVENTION OF OTO-PATHOLOGIES BY INHIBITION OF PRO-APOPTOTIC GENES - The invention relates to one or more inhibitors, in particular siRNAs, which down-regulate the expression of human pro-apoptotic genes. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating or preventing the incidence or severity of hearing impairment (or balance impairment), particularly hearing impairment associated with cell death of the inner ear hair cells or outer ear hair cells, comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient.07-25-2013
20130190380ANTISENSE OLIGONUCLEOTIDES DIRECTED AGAINST CONNECTIVE TISSUE GROWTH FACTOR AND USES THEREOF - This invention provides compounds which comprise modified oligonucleotides capable of inhibitory expression of connective tissue factor and composition containing same as well as methods of treating hyperprolific disorders and fibrotic diseases, and of reducing scarring resulting from wound healing using such compounds.07-25-2013
20130190379SMALL RNA MOLECULES, PRECURSORS THEREOF, MEANS AND METHODS FOR DETECTING THEM, AND USES THEREOF IN TYPING SAMPLES - The invention is related to small RNA molecules, precursors thereof and methods for detecting such molecules. The invention is in particular concerned with differentially expressed small RNA molecules and precursors thereof. Various collections of small RNA molecules, precursors thereof and collections of probe and primers that can be used to detect small RNA molecules, precursors thereof are provided. Further provided are diagnostic test based on this differential expression. Also provided are therapeutic applications using one or more of the members of the presented collections.07-25-2013
20110118331CATIONIC SIRNAS, SYNTHESIS AND USE FOR RNA INTERFERENCE - The invention relates to cationic siRNAs, characterized in that they are double-stranded RNA fragments, grafted to the ends of which are oligocations, the number of cationic charges grafted being comparable to or greater than that of the anionic charges carried by the internucleoside phosphates of the RNA strands.05-19-2011
20110118335RNA Interference Mediated Inhibition Of Gene Expression Using Multifunctional Short Interfering Nucleic Acid (Multifunctional siNA) - The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.05-19-2011
20110118338METHODS, COMPOSITIONS AND COMPOUND ASSAYS FOR INHIBITING AMYLOID-BETA PROTEIN PRODUCTION - A method for identifying compounds that inhibit amyloid-beta precursor protein processing in cells, comprising contacting a test compound with a GPCR polypeptide, or fragment thereof, and measuring a compound-GPCR property related to the production of amyloid-beta peptide. Cellular assays of the method measure indicators including second messenger and/or amyloid beta peptide levels. Therapeutic methods, and pharmaceutical compositions including effective amyloid-beta precursor processing-inhibiting amounts of GPCR expression inhibitors, are useful for treating conditions involving cognitive impairment such as Alzheimers Disease.05-19-2011
20110124710COMPOSITION AND METHODS OF RNAI THERAPEUTICS FOR TREATMENT OF CANCER AND OTHER NEOVASCULARIZATION DISEASES - Compositions and methods are provided for treatment of diseases involving unwanted neovascularization (NV). The invention provides treatments that control NV through selective inhibition of pro-angiogenic biochemical pathways, including inhibition of the VEGF pathway gene expression and inhibition localized at pathological NV tissues. Tissue targeted nanoparticle compositions comprising polymer conjugates and nucleic acid molecules that induce RNA interference (RNAi) are provided. The nanoparticle compositions of the invention can be used alone or in combination with other therapeutic agents such as VEGF pathway antagonists. The compositions and methods can be used for the treatment of NV diseases such as cancer, ocular disease, arthritis, and inflammatory diseases.05-26-2011
20110124707Methods And Compositions For Reducing Viral Genome Amounts In A Target Cell - Methods and compositions for reducing viral genome amounts in a target cell are provided. In the subject methods, the activity of a miRNA is inhibited in a manner sufficient to reduce the amount of viral genome in the target cell, e.g., by introducing a miRNA inhibitory agent in the target cell. Also provided are pharmaceutical compositions, kits and systems for use in practicing the subject methods. The subject invention finds use in a variety of applications, including the treatment of subjects suffering from a viral mediated disease condition, e.g., an HCV mediated disease condition.05-26-2011
20110124711COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Nav1.8 GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Nav1.8 gene (Nav1.8 gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene in a cell.05-26-2011
20110124709RNA ANTAGONISTS TARGETING GLI2 - The present invention relates to oligomer compounds (oligomers), which target GLI2 mRNA in a cell, leading to reduced expression of GLI2. Reduction of GLI2 expression is beneficial for the treatment of certain medical disorders, such as hyperproliferative disorders, such as cancer.05-26-2011
20110124708RNAI-MEDIATED INHIBITION OF OCULAR HYPERTENSION TARGETS - RNA interference is provided for inhibition of ocular hypertension target mRNA expression for lowering elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. Ocular hypertension targets include carbonic anhydrase II, IV, and XII; β1- and β2 adrenergic receptors; acetylcholinesterase; Na05-26-2011
20110086901OLIGONUCLEOTIDES AFFECTING EXPRESSION OF PHOSPHODIESTERASES - The invention relates to therapeutic antisense oligonucleotides directed against genes encoding phosphodiesterases (PDE) and the use of these antisense oligonucleotides in combination. These antisense oligonucleotides may be used as analytical tools and/or as therapeutic agents in the treatment of disease associated with reduced cellular cAMP in a patient, such as inflammatory diseases of the respiratory tract including, for example, asthma, chronic obstructive pulmonary disease (COPD), acute respiratory distress syndrome, bronchitis, chronic bronchitis, silicosis, pulmonary fibrosis, lung allograft rejection, allergic rhinitis and chronic sinusitis as well as other conditions in which an increase in cyclic AMP or a decrease in PDE levels is beneficial.04-14-2011
20120035242PHARMACEUTICAL COMPOSITION FOR TREATING OBESITY OR DIABETES - The invention is intended to provide a pharmaceutical composition for treating or preventing obesity or type II diabetes and a method for treatment for them. A pharmaceutical composition comprising an Acsl-1 inhibitor of this invention as an active ingredient is useful for prevention or treatment of obesity or type II diabetes.02-09-2012
20110082185CANCER-TESTIS GENE SILENCING AGENTS AND USES THEREOF - The invention relates to methods, formulations and kits useful for inhibiting cancer cell viability, invasion, or migration.04-07-2011
20110082186COMPOSITIONS FOR INHIBITING GENE EXPRESSION AND USES THEREOF - The inventors have examined the means for providing more efficacious gene expression blocking compounds. The inventors have discovered new structural features that surprisingly improve the efficacy of gene expression blocking molecules. These features include the presence of multiple 3′ ends and a linker at the 5′ ends. Surprisingly, these features improve the efficacy of the gene expression blocking compounds in a manner that decreases the compound's biologic instability. Even more surprisingly, this effect has been found to be applicable to both DNA and RNA oligonucleotide-based compounds and to have application in traditional antisense and RNAi technologies.04-07-2011
20110213013Complexes of Small-Interfering Nucleic Acids - The present invention relates to complexes of small-interfering nucleic acids (siNA). Compositions of siNA suited for administration to a patient are described. Methods for delivering the compositions are also described.09-01-2011
20090247606RNA Interference Mediated Inhibition of Adenosine A1 Receptor (ADORA1) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention concerns methods and reagents useful in modulating adenosine A1 receptor (ADORA1) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small interfering RNA (siRNA) molecules capable of mediating RNA interference (RNAi) against ADORA1 and related receptors.10-01-2009
20100227911RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating chromosomal translocation gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of chromosomal translocation gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of BCR-ABL, ERG, EWS-ERG, TEL-AML1, EWS-FLI1, TLS-FUS, PAX3-FKHR, and/or AML1-ETO fusion genes.09-09-2010
20110245324ANTIMICROBIAL COMPOUNDS - The present invention relates to compounds that modulate the shikimate pathway and/or a pathway branching from the shikimate pathway in members of the Amoebida Order. In particular these compounds may be useful in the treatment or prevention of diseases caused or contributed to by members of the Amoebida Order.10-06-2011
20100197763RNA Interference Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase (PDE4B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-7, IL-8, IL-15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP-2, MMP-3, MMP-9 and MMP-12. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC) including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.08-05-2010
20100069461COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF FACTOR V LEIDEN MUTANT GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor V Leiden mutant gene (Factor V Leiden mutant gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Factor V Leiden mutant gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Factor V Leiden mutant gene using the pharmaceutical composition; and methods for inhibiting the expression of the Factor V Leiden mutant gene in a cell.03-18-2010
20110251257METHODS AND COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE - This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases.10-13-2011
20090312395Assay - RAN and RAN Binding Protein 1 have been determined to be markers of invasive and metastatic potential of a tumour cell. There is described methods and kits for the detection of the level of RAN and RAN Binding Protein 1 and the use thereof.12-17-2009
20090312394Protection against and treatment of age related macular degeneration - Methods and reagents in relation to the diagnosis, protection and treatment of Age Related Macular Degeneration (AMD). In particular, the methods and reagents in relation to RNA; determined to provide a strong protection to a subject against development of AMD.12-17-2009
20100016407Combined Telomerase Inhibitor and Gemcitabine for the Treatment of Cancer - A method and kit for inhibiting the proliferation of cancer cells are disclosed, based on a combination of a gemcitabine and a telomerase inhibitor. When used in cancer therapy, the two compounds in combination enhance the anti-cancer treatment efficacy obtained with gemcitabine alone or the telomerase inhibitor alone. Preferably, efficacy is supraadditive or synergistic in nature relative to the combined effects of the individual agents, with minimal exacerbation of side effects.01-21-2010
20110152350Compositions and Methods for Inhibiting Expression of XBP-1 Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting X-Box Protein 1 (XBP-1), and methods of using the dsRNA to inhibit expression of XBP-1.06-23-2011
20110152356NOVEL RNAi THERAPEUTIC FOR TREATMENT OF HEPATITIS C INFECTION - Small interfering RNAs (siRNAs) or small hairpin RNA (shRNAs) and compositions comprising same are provided that specifically target human cyclophilin A (CyPA) to effectively inhibit Hepatitis C(HCV) infection in a cell. Such siRNA and shRNAs may have a length of from about 19 to about 29 contiguous nucleotides corresponding to a specific region of human cyclophilin A (CyPA) cDNA of from about nucleotide 155 to about nucleotide 183 having particular potency against CyPA and HCV. Such siRNA and shRNAs may be formulated as naked compositions or as pharmaceutical compositions. DNA polynucleotides, plasmids, and viral or non-viral vectors are also provided that encode siRNA or shRNA molecules, which may be delivered directly to cells or in combination with known delivery agents, such as lipids, polymers, encapsulated lipid particles, such as liposomes. Methods for treating, managing inhibiting, preventing, etc., HCV infection using such siRNA and shRNAs and compositions comprising same are also provided.06-23-2011
20100056608METHODS FOR SCREENING AND TREATMENT INVOLVING THE GENES GYPC, AGPAT3, AGL, PVRL2, HMGB 3, HSDL2 AND/OR LDB2 - The present invention relates to a method for identifying a compound as a candidate drug, comprising the steps a. bringing said compound into contact with a cell expressing the genes CYPC, AGPAT3, AGL, PVRL2, HMGB 3, HSDL2; and b. analyzing if said compound modulates the expression of at least one of said genes. It also relates to a method for identifying a compound as a candidate drug, comprising the steps a. bringing said compound into contact with a cell expressing the gene LDB2; and b. analyzing if said compound modulates the expression of LDB2. The invention further relates to genetically modified cells and animals useful in such methods and to methods for treatment of atherosclerosis, atherosclerosis-related diseases or inflammatory diseases, comprising the use of such identified compounds.03-04-2010
20100056609METHODS AND COMPOSITIONS FOR INHIBITION OF AXONAL DEGENERATION BY MODULATION OF THE DLK/JNK PATHWAY - Methods of reducing Wallerian degeneration are disclosed. These methods comprise inhibiting expression or activity of a mixed lineage kinas such as a dual leucine-zipper-bearing kinase (DLK), inhibiting expression or activity of a molecule acting downstream from DLK, such as a c-Jun N-terminal kinase (JNK), or a combination thereof. Further disclosed are methods of screening candidate compounds for DLK inhibition activity. These methods comprise providing a neuronal culture comprising a plurality of axons; contacting the culture with a candidate compound and with an axon degeneration-triggering agent; and comparing axonal degeneration in the culture to a control culture comprising the axon degeneration-triggering agent but not the candidate compound.03-04-2010
20100048676Non-androgen dependent roles for androgen receptor in liver cancer - Disclosed are compositions and methods for modulating AR activity, such as non-androgen dependent AR activity. Also disclosed are compositions and methods for diagnosing beast cancer and for inhibiting liver cancer growth. In addition, disclosed are methods for identifying molecules that inhibit AR in non-androgen dependent ways.02-25-2010
20100048675miRNA TRIPLEX FORMATIONS FOR THE DOWNREGULATION OF VIRAL REPLICATION - As discovered herein, using miRNAs having high homology with both HIV-02-25-2010
20100069465METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.03-18-2010
20100069463METHODS FOR THE DIAGNOSIS AND TREATMENT OF AFFECTIVE DISORDERS AND CUSHING'S SYNDROMES - The present invention relates to a method for identifying a TMEFF2 modulator, comprising (a) contacting a cell which expresses TMEFF2 with a candidate compound to be tested; (b) measuring whether said compound to be tested decreases or increases the level of a constituent of the cAMP signalling pathway, preferably the CRH signalling pathway, in said cell when compared to a corresponding cell which does not express TMEFF2; (b′) optionally determining whether said compound is capable of reduncing the binding between Activin and TMEFF2; and (c) identifying said modulator compound. Furthermore, a method for identifying a TMEFF2 modulator comprising determining whether said TMEFF2 modulator is capable of reducing the binding between Activin and TMEFF2 is contemplated. It also relates to uses and methods applying a TMEFF2 agonist for the treatment of Cushing's Syndromes and a TMEFF2 modulator for the treatment of affective disorders. Furthermore, methods of diagnosing affective disorders or Cushing's Syndromes are provided.03-18-2010
20110178160Inhibition of Interaction of PSD93 and PSDS95 with nNOS and NMDA Receptors - PSD-95/SAP90 antisense-treated animals not only experience a significant decrease in MAC for isoflurane, but also experience an attenuation in the NMDA-induced increase in isoflurane MAC. PSD-95/SAP90 appears to mediate the role of the NMDA receptor in determining the MAC of inhalational anesthetics. Suppression of the expression of PSD-95/SAP90 in the spinal cord significantly attenuates responses to painful stimuli mediated through the N-methyl-D-aspartate receptor activation. In spinal cord neurons PSD-95/SAP90 interacts with the N-methyl-D-aspartate receptor subunits 2A/2B. Activation of the N-methyl-D-aspartate receptor in spinal hyperalgesia results in association of the N-methyl-D-aspartate receptor with PSD-95/SAP90. PSD-95/SAP90 is required for hyperalgesia triggered via the N-methyl-D-aspartate receptor at the spinal cord level.07-21-2011
20110178158Set of Antiangiogenic Molecules and Use Thereof - The present invention relates to an antiangiogenic pharmaceutical composition comprising at least one molecule selected from the group comprising an antisense nucleic acid molecule of VEGF 165, an antisense nucleic acid molecule of alpha-9 domain that forms integrin alpha-9/beta-1 and an antisense nucleic acid molecule of VAP-1, a molecule of VEGF 165-specific antibody, a molecule of antibody specific for the alpha-9 domain that forms integrin alpha-9/beta-1 and a molecule of VAP-1-specific antibody and/or combinations thereof and a pharmaceutically acceptable vehicle. The present invention also protects the use of said molecules for the production of a medicament.07-21-2011
20110178154 GENE EXPRESSION PROFILE THAT PREDICTS OVARIAN CANCER SUBJECT RESPONSE TO CHEMOTHERAPY - A gene profiling signature is disclosed herein. The gene signature can predict whether a subject with ovarian cancer will be chemorefractory, chemoresistant or chemosensitive. Thus, methods are disclosed for determining whether a subject with ovarian cancer is sensitive to treatment with a chemotherapeutic agent. Methods are also provided for increasing sensitivity to the chemotherapeutic agent if the presence of differential expression indicates that the ovarian cancer has a decreased sensitivity to chemotherapeutic agent.07-21-2011
20110178153METHODS OF TREATING CHEMORESISTANCE AND RELAPSE IN CANCER CELLS - Methods of treating or preventing chemoresistance or relapse growth of cancer cells are provided. Methods of treating or preventing resistance to tyrosine kinase based chemotherapeutic treatment in hematologic and solid tumors are provided. BCR-ABL drug resistance in chronic myelogenous leukemia (CML) and models for conducting further study on the same are presented. The methods comprise administering a therapeutically effective amount of one or more SIRT1 modulators to the cells or subject in need thereof. The methods may be administered in combination with, prior to or subsequent to chemotherapy or may be administered to counteract the effect of a spontaneous genetic mutation. Methods of using SIRT1 inhibitors to treat or prevent insulin and transferrin-induced resistance are also presented. A novel cell model to study mechanisms of acquired chemoresistance is also provided.07-21-2011
20100056607COMPOSITIONS AND THEIR USES DIRECTED TO HBXIP - Disclosed herein are compounds, compositions and methods for modulating the expression of HBXIP in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.03-04-2010
20100056610HEPTbeta AS A TARGET IN TREATMENT OF ANGIOGENISIS MEDIATED DISORDERS - Disclosed herein is the use of HPTPbeta to screen agents useful in the treatment angiogenesis mediated disorders.03-04-2010
20090215865Nucleic Acid Molecules and Collections Thereof, Their Application and Identification - The invention provides a method for characterising a sample comprising nucleic acid derived from a cell. The method comprises determining whether a sample comprises at least a minimal sequence of at least one new microRNA (miRNA) according to the invention or a mammalian ortholog thereof and characterizing the sample on the basis of the presence or absence of the miRNA. The invention further provides new nucleic acid molecules and collections thereof and their use in therapeutic and diagnostic applications. The invention furthermore provides a method for identifying a miRNA molecule or a precursor molecule thereof.08-27-2009
20090215863Gene Silencing of Protease Activated Receptor 1(Par1) - The present invention relates to nucleic acid molecules, vectors, compositions, and methods useful for modulating protease-activated receptor 1 gene expression via RNA interference. In particular, the instant invention features small interfering RNA (siRNA) and short hairpin RNA (shRNA) molecules and methods for modulating the expression of protease-activated receptor 1 gene.08-27-2009
20090215864Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer - The invention provides novel double stranded oligoribonucleotides that inhibit the Nrf2 gene. The invention also provides a pharmaceutical composition comprising one or more such oligoribonucleotides, and a vector capable of expressing the oligoribonucleotide. The present invention also relates to methods and compositions for treating or preventing the incidence or severity of a cancerous disease, particularly various lung cancers.08-27-2009
20090215859Compositions and methods for modulating dhr96 - Disclosed are compositions and methods for modulating DHR96 activity and identifying molecules that modulate DHR96 activity.08-27-2009
20110152353DOUBLE-STRANDED POLYNUCLEOTIDE - It is intended to provide a double-stranded polynucleotide that is resistant to RNase and has RNA interference effect, etc. The present invention provides a double-stranded polynucleotide comprising sense and antisense strands comprising polynucleotides comprising a nucleotide unit of DNAs and 2′-O-methyl RNAs alternately combined.06-23-2011
20110152351MODULATION OF SMRT EXPRESSION - Disclosed herein are compounds and methods for decreasing SMRT and treating metabolic and/or cardiovascular diseases in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to SMRT include obesity, diabetes, dyslipidemia, and hypothyroidism.06-23-2011
20110086903EMERGENCE OF A R-TYPE CA2+ CHANNEL (CAV 2.3) CONTRIBUTES TO CEREBRAL ARTERY CONSTRICTION FOLLOWING SUBARACHNOID HEMORRHAGE - The invention relates to methods and products for treatment of a neurological defect such as a subarachnoid hemorrhage or cerebral vasospasm. Specifically, R-type voltage-gated calcium channel inhibitors and related compositions and kits are described.04-14-2011
20100069466Compositions and Methods for the Systemic Treatment of Arthritis - The present invention includes compositions and methods for treating arthritic joints found in patients with autoinflammation, e.g., systemic onset juvenile idiopathic arthritis, by administering at the site of inflammation a therapeutically effective amount of at least one agent that reduces or blocks the bioavailability of interleukin-1β.03-18-2010
20100069464COMPOSITIONS AND METHODS FOR MODULATING ACTIVITY OF CAPPED SMALL RNAS - Compositions and methods for modulating transcription by RNA polymerases are described.03-18-2010
20110098343Antisense Formulation - A room temperature stable and minimal aggregate liquid formulation comprises an oligonucleotide comprising Seq ID No. 1: or comprising a variant oligonucleotide in which no more than 3 non-sequential bases are different from Seq. ID NO. 1 and an aqueous carrier comprising a aggregation-preventing compound selected from the group consisting of mono and disaccharides and/or sugar alcohols.04-28-2011
20110251263RNAi Modulation of the BCR-ABL Fusion Gene and Uses Thereof - The invention relates to compositions and methods for modulating the expression of Bcr-Abl, and more particularly to the down-regulation of Bcr-Abl mRNA and Bcr-Abl protein levels by oligonucleotides via RNA interference, e.g., chemically modified oligonucleotides.10-13-2011
20110251262Compositions And Methods For Inhibiting Expression Of An RNA From West Nile Virus - This invention relates to double-stranded ribonucleic acid (dsRNA), and its use in mediating RNA interference to inhibit the expression of an RNA from the West Nile virus (WNV), and the use of the dsRNA to treat pathological processes mediated by WNV infection, such as viral encephalitis.10-13-2011
20110251261COMPOSITIONS AND METHODS FOR INHIBITION OF NUCLEIC ACIDS FUNCTION - The invention relates generally to compositions and methods for inhibiting the function of target nucleic acids by sequence specific binding. The compositions and methods can be used for inhibition of micro RNAs and other relatively short non-coding RNAs.10-13-2011
20110251255Methods and Compositions for Inhibiting the Proliferation of Cancer Cells - A method of decreasing the expression of LIM kinase 1 in a cancer cell comprising; providing an oligonucleotide consisting of the sequence of SEQ ID NO: 1; providing a cancer cell comprising an mRNA encoding LIM kinase 1; and introducing the oligonucleotide into the cancer cell, wherein the oligonucleotide decreases the expression of LIM kinase 1 in the cancer cell. The method also provides compositions of an antisense RNA LIM kinase 1 that can be administered to an individual for the purpose of inhibiting a protein kinase pathway and which further comprises methods for treating and monitoring the proliferation and metastasis of cancer cells. A kit may be used in the detection and treatment of cancer.10-13-2011
20110098342METHODS OF DETECTING LONG RANGE CHROMOSOMAL INTERACTIONS - The present invention relates to a method of monitoring epigenetic changes comprising monitoring changes in conditional long range chromosomal interactions at at least one chromosomal locus where the spectrum of long range interaction is associated with a specific physiological condition, the method comprising the steps of: —(i) in vitro crosslinking of said long range chromosomal interactions present at the at least one chromosomal locus; (ii) isolating the cross linked DNA from said chromosomal locus; (iii) subjecting said cross linked DNA to restriction digestion with an enzyme that cuts at least once within the at least one chromosomal locus; (iv) ligating said cross linked cleaved DNA ends to form DNA loops; (v) identifying the presence of said DNA loops; wherein the presence of DNA loops indicates the presence of a specific long range chromosomal interaction.04-28-2011
20110098341USE OF ID4 FOR DIAGNOSIS AND TREATMENT OF CANCER - The invention relates to a method of determining whether a human subject is suffering from or at risk for developing pancreatic cancer by determining the methylation level of an ID4 gene promoter or the expression level of an ID4 gene in a biological sample from a human subject. Also disclosed are a method of analyzing the methylation level of an ID4 gene promoter or the expression level of an ID4 gene in a pancreatic cancer cell, and a method of inhibiting the methylation of an ID4 gene promoter or enhancing the expression of an ID4 gene by contacting a pancreatic cancer cell with a compound that decreases the methylation level of an ID4 gene promoter or increases the expression level of an ID4 gene in the cell.04-28-2011
20110098340ANTISENSE MODULATION OF BCL2-ASSOCIATED X PROTEIN EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of BCL2-associated X protein. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding BCL2-associated X protein. Methods of using these compounds for modulation of BCL2-associated X protein expression and for treatment of diseases associated with expression of BCL2-associated X protein are provided.04-28-2011
20110098339C2ORF18 AS TARGET GENE FOR CANCER THERAPY AND DIAGNOSIS - Described herein are objective methods for detecting or diagnosing a predisposition to developing cancer, particularly pancreatic cancer. In one embodiment, the diagnostic method involves the step of determining an expression level of C2orf18 using anti-C2orf18 antibody. The present invention further provides methods of screening for therapeutic agents useful in the treatment of a C2orf18-associated disease, such as a cancer, e.g. pancreatic cancer, methods of inhibiting the cell growth and treating or alleviating their symptom. The invention also features products, such as polynucleotides, polypeptides, and vectors double-stranded molecules, antibodies, vectors and compositions composed thereof.04-28-2011
20110098338RNA Interference for the Treatment of Heart Failure - The present invention relates to targeted RNAi for the treatment of heart failure by modulating defective cardiac Ca04-28-2011
20110098337Inhibitors of RTP801 and their use in disease treatment - The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases respiratory conditions and hearing disorders based upon inhibition of the RTP801 gene and/or protein.04-28-2011
20110077283MOLECULAR TARGETS AND COMPOUNDS, AND METHODS TO IDENTIFY THE SAME, USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES - The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant huntingtin protein, inhibiting or reducing polyglutamine-induced protein aggregation, and/or altering huntingtin protein conformation, which inhibition is useful in the prevention, amelioration and/or treatment of neurodegenerative diseases, and protein aggregation diseases more generally. In particular, the present invention provides methods and assays for identifying agents for use in the prevention and/or treatment of Huntington's disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA sequences based on these03-31-2011
20110082188GENE EXPRESSION PROFILING OF INFLAMMATORY BOWEL DISEASE - The present invention relates to methods for identifying and/or classifying patients with inflammatory bowel diseases (IBD), particularly patients with Crohn's disease or ulcerative colitis. Gene expression profiling shows broad and fundamental differences in the pathogenic mechanism of UC and CD. The subject method is based on the findings that certain genes are differentially expressed in intestinal tissue of IBD patients compared with related normal cells, such as normal colon cells. That change can be used to identify or classify IBD cells by the upregulation and/or downregulation of expression of particular genes, alterations in protein levels or modification, or changes at the genomic level (such as mutation, methylation, etc), e.g., an event which is implicated in the pathology of inflammatory bowel diseases.04-07-2011
20110082187MARKERS AND METHODS RELATING TO THE ASSESSMENT OF ALZHEIMER'S DISEASE - Use of clusterin as a biomarker of Alzheimer's disease (AD), particularly methods and compositions for detection of clusterin in a biological sample and assessment of in vivo pathology, disease severity and rate of clinical progression in a subject having or suspected of having AD.04-07-2011
20110071211MicroRNA (miRNA) And Downstream Targets For Diagnostic And Therapeutic Purposes - The present invention relates to a promoter region of a microRNA, the use of a microRNA, in particular miR-21, and related elements for the diagnosis and for the manufacture of a medicament for the treatment and/or prevention of fibrosis and/or fibrosis related diseases. Additionally, the invention concerns antisense oligonucleotides against targets of miR-21. A cell deficient for miR-21, the promoter region and targets of miR-21 and a knock-out organism thereof are also encompassed. Finally, the invention is directed to a method for diagnosing fibrosis and/or fibrosis related diseases and to a method for screening a pharmaceutically active compound for the treatment of fibrosis and/or fibrosis related diseases. The present invention further relates to compositions for use in the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions modulate the activity of a miRNA for the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions inhibit the activity of miR-21 for the treatment, amelioration, and/or prevention of fibrosis.03-24-2011
20110152357Micro-RNA-Based Compositions and Methods for the Diagnosis, Prognosis and Treatment of Multiple Myeloma - Methods for assessing a pathological condition in a subject includes measuring an expression profile of one or more markers where a difference is indicative of multiple myeloma (MM) or a predisposition to MM. The present invention provides novel methods and compositions for the diagnosis, prognosis and treatment of MM. The invention also provides methods of identifying anti-MM cancer agents.06-23-2011
20110152354Bispecific Oligonucleotide for the Treatment of CNS Malignancies - CNS malignancy is treated in a subject suffering from a CNS malignancy by administering to the subject an antisense oligonucleotide having a sequence of bases that is complementary to portions of both the gene encoding IGFBP-2 and the gene encoding IGFBP-5, and which is of sufficient length to act as an inhibitor of the effective amount of IGFBP-2 and IGFBP-5, in an amount effective to reduce effective levels of IGFBP-2 and IGFBP-5 in cells of the CNS malignancy.06-23-2011
20110152348METHODS FOR TREATING ANDROGEN RECEPTOR DEPENDENT DISORDERS INCLUDING CANCERS - The invention provides the combination use of antisense oligomers targeting androgen receptor mRNA and androgen receptor binding inhibitors that reduce androgen receptor activity for the treatment of androgen receptor related medical disorders, such as cancers, particularly prostate cancers and breast cancers.06-23-2011
20110077285RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF PIK3CA EXPRESSION - The invention relates to oligomeric compounds (oligomers), which target PIK3CA mRNA in a cell, leading to reduced expression of PIK3CA. Reduction of PIK3CA expression is beneficial for the treatment of certain medical disorders, such as hyperproliferative diseases (e.g., cancer). The invention provides therapeutic compositions that include the oligomers and methods for modulating the expression of PIK3CA using the oligomers, including methods of treatment.03-31-2011
20110077284DRY POWDER COMPOSITIONS FOR RNA INFLUENZA THERAPEUTICS - A dry powder formulation for delivery to a mammal by inhalation, the formulation comprising particles comprising a lipid, a carrier, and one or more double-stranded siRNA molecules or dicer-active precursors targeted to influenza virus A method for treating or preventing influenza in a mammal comprising administering a therapeutically-effective amount of a dry powder formulation.03-31-2011
20110071209RNAi Modulation of the RHO-A Gene and Uses Thereof - The invention relates to compositions and methods for modulating the expression of the RhoA gene, and more particularly to the downregulation of RhoA by chemically modified oligonucleotides.03-24-2011
20110071208LIPID ENCAPSULATED DICER-SUBSTRATE INTERFERING RNA - The present invention provides novel, stable nucleic acid-lipid particles comprising one or more Dicer-substrate dsRNAs and/or small hairpin RNAs (shRNAs), methods of making the particles, and methods of delivering and/or administering the particles (e.g., for the treatment of a disease or disorder). In some embodiments, the nucleic acid-lipid particles of the invention comprise Dicer-substrate dsRNAs and/or shRNAs. In other embodiments, the nucleic acid-lipid particles of the invention comprise Dicer-substrate dsRNAs and/or shRNAs in combination with one or more additional interfering RNAs (e.g., siRNA, aiRNA, and/or miRNA). In further embodiments, the Dicer-substrate dsRNAs and/or shRNAs present in the nucleic acid-lipid particles of the invention are chemically synthesized.03-24-2011
20120149760DISABLING AUTOPHAGY AS A TREATMENT FOR LYSOSOMAL STORAGE DISEASES - Provided herein are methods of treating lysosomal storage disease, for instance Pompe disease, through inhibition of autophagy. Optionally, treatment is administered as an adjunct to enzyme replacement therapy (ERT).06-14-2012
20120149757COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING - Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.06-14-2012
20120035245METHODS FOR IDENTIFYING AND COMPOUNDS USEFUL FOR THE DIAGNOSIS AND TREATMENT OF DISEASES INVOLVING INFLAMMATION - The present invention relates to agents, and methods for identifying compounds, which agents and compounds result in the stabilization of mast cells, in particular that inhibit mast cell degranulation. In addition, the invention relates to compositions and methods for the use thereof in treating conditions that are characterized by mast cell degranulation and/or inflammation, including allergic rhinitis.02-09-2012
20110065773RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a 03-17-2011
20120202873RNAi Probes Targeting Cancer-Related Proteins - RNAi sequences that are useful as therapeutics in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma and melanoma; and Alzheimer's disease. These sequences target clusterin, IGFBP-5, IGFBP-2, both IGFBP-2 and -5 simultaneously, Mitf, and B-raf. The invention further provides for the use of these RNAi sequences in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma and melanoma; and Alzheimer's disease, and a method of treating such conditions through the administration of the RNA molecules with RNAi activity to an individual, including a human individual in need of such treatment.08-09-2012
20110251259COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF CD274/PD-L1 GENE - The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the CD274/PD-L1 gene, and methods of using such dsRNA compositions to inhibit expression of CD274/PD-L1.10-13-2011
20110251258RNAI CONSTRUCTS AND USES THEREOF - The invention relates to improved double-stranded RNAi constructs (sometimes referred to as “solo-rxRNA”) and uses thereof. The construct comprises a structure formed in some aspects of the invention by two identical single-stranded polynucleotides, with the structure having two double-stranded stem regions (each having less than 21 base pairs) and a loop or bulge having about 4 to 11 nucleotides on each strand. The construct is resistant to cleavage by Dicer or other Dicer-like RNase III enzymes and is capable of being loaded into a RISC complex to effect RNA interference. In addition, the nucleotides of the present hairpin constructs may be modified to greatly enhance functionality, such as stability and specificity.10-13-2011
20120202874ANTISENSE OLIGONUCLEOTIDE MODULATION OF STAT3 EXPRESSION - Compounds, compositions and methods are provided for inhibiting the expression of human STAT3. The compositions comprise antisense oligonucleotides targeted to nucleic acids encoding STAT3. Methods of using these oligonucleotides for inhibition of STAT3 expression and for promotion of apoptosis are provided. Methods for treatment of diseases, particularly inflammatory diseases and cancers, associated with overexpression or constitutive activation of STAT3 or insufficient apoptosis are also provided.08-09-2012
20110054006REGULATION OF ONCOGENES BY MICRORNAS - Naturally occurring miRNAs that regulate human oncogenes and methods of use thereof are described. Suitable nucleic acids for use in the methods and compositions described herein include, but are not limited to, pri-miRNA, pre-miRNA, mature miRNA or fragments of variants thereof that retain the biological activity of the mature miRNA and DNA encoding a pri-miRNA, pre-miRNA, mature miRNA, fragments or variants thereof, or regulatory elements of the miRNA. The compositions are administered to a subject prior to administration of a cytotoxic therapy in an amount effective to sensitize cells or tissues to be treated to the effects of the cytotoxic therapy.03-03-2011
20110009473PTTG1 AS A BIOMARKER FOR CANCER TREATMENT - The present invention relates to the use of pituitary tumor transforming gene 1 (PTTG1) as a biomarker for diagnosing cancer as well as for determining cancer treatment responsiveness. In one embodiment, the present invention provides a method of treating cancer by inhibiting the expression of PTTG1, and administering a therapeutically effective amount of Aurora kinase inhibitor, HDAC inhibitor and/or ROS-generating agent.01-13-2011
20100267805TARGETING OPPOSITE STRAND REPLICATION INTERMEDIATES OF SINGLE-STRANDED VIRUSES BY RNAI - The invention relates to methods and compositions for modulating viral replication through double-stranded RNA-mediated gene silencing (RNAi), wherein the antiviral methods and compositions preferentially target opposite strand replication intermediates of single-stranded RNA viruses.10-21-2010
20100120895RNA Interference Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase (PDE4B) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-7, IL-8, IL-15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP-2, MMP-3, MMP-9 and MMP-12. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC) including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.05-13-2010
20100120891USE OF A GALECTIN-1-TARGETED RNAI-BASED APPROACH FOR THE TREATMENT OF CANCER - The present invention relates to an RNAi molecule suitable for reducing the expression of galectin-1 containing any of the sequences of SEQ ID NOs: 1-33, and preferably the sequences of SEQ ID NO: 2, 3, or 4, and to the use thereof as a medicament, or for the manufacture of a medicament for treating and/or for delaying the progression of cancer, preferably glioma, pancreatic cancer, head and neck cancer, melanoma, non-small-cell lung cancer and non-Hodgkin's lymphoma. The present invention also relates to compositions and methods for treating and for delaying the progression of cancer, preferably glioma, pancreatic cancer, head and neck cancer, melanoma, non-small-cell lung cancer and non-Hodgkin's lymphoma, for reducing the migration of tumor cells, preferably cells of glioma, pancreatic cancer, head and neck cancer, melanoma, non-small-cell lung cancer and non-Hodgkin's lymphoma, and/or for enhancing the efficacy of cancer therapies for the treatment of cancer, preferably glioma, pancreatic cancer, head and neck cancer, melanoma, non-small-cell lung cancer and non-Hodgkin's lymphoma, selected from the group comprising chemotherapy, radiation therapy, immunotherapy, and/or gene therapy.05-13-2010
20110152346Use of Oligonucleotides with Modified Bases in Hybridization of Nucleic Acids - The invention is concerned with the use of oligonucleotide analogs that contain specifically modified DNA bases to be used in hybridization of nucleic acids, polymerase chain reaction (PCR) and siRNA-mediated gene silencing (RNAi).06-23-2011
20100280095THERAPEUTIC AGENT FOR WOUNDS AND SCREENING METHOD FOR THE SAME - The present invention provides an agent for treating wound, containing a substance that suppresses the expression or function of PDLIM2. The substance that suppresses the expression or function of PDLIM2 is preferably an siRNA or antisense nucleic acid capable of specifically suppressing the expression of PDLIM2, or an expression vector capable of expressing said polynucleotide. In addition, the present invention provides a method of screening for a substance capable of treating wounds, including determining whether or not a test substance is capable of suppressing the expression or function of PDLIM2.11-04-2010
20110213011MODULATION OF SMAD3 EXPRESSION - Provided are compounds capable of inhibiting SMAD3 and compositions containing same as well as methods using such compounds for treating fibrosis and scarring.09-01-2011
20110213009ADFP MODULATORS IN THE TREATMENT OF ACNE, OF SEBORRHOEIC DERMATITIS OR OF HYPERSEBORRHOEA - An in vitro or in vivo method for screening for candidate compounds for the preventive or curative treatment of acne, of seborrhoeic dermatitis or of skin disorders associated with hyperseborrhoea, includes determining the ability of a compound to modulate the expression or the activity of the adipose differentiation-related protein (ADFP), and also utilizes modulators of the expression or of the activity of this protein, for the treatment of acne, of seborrhoeic dermatitis or of skin disorders associated with hyperseborrhoea; methods for the in vitro diagnosis of or in vitro prognosis for these pathologies are also featured.09-01-2011
20110160281RNA Interference Mediated Inhibition of Vascular Cell Adhesion Molecule (VCAM) Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating vascular cell adhesion molecule gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of vascular cell adhesion molecule gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of vascular cell adhesion molecule genes, such as vascular cell adhesion molecule-1 (VCAM-1).06-30-2011
20110178156Method of suppressing pRb deficiency-induced tumor formation - The present invention provides methods of determining a putative agent that inhibits tumorigenesis in retinoblastoma protein deficient cells, the methods comprising determining whether the putative agent decreases phosphorylation of threonine residue 187 of p27, decreases S-phase kinase-associated protein 2 interaction with p27 having a phosphorylated threonine residue 187, or an increase in apoptosis of retinoblastoma protein deficient cells. The present invention also provides the agent, the pharmaceutical composition, and methods of inhibiting, preventing and treating tumorigenesis in retinoblastoma deficient cells, the method comprising administration of the agent that decreases phosphorylation of threonine residue 187 of p27 or the S-phase kinase-associated protein 2 interaction with p27 having a phosphorylated threonine residue 187.07-21-2011
20120202875Method Of Treatment - The present invention relates to methods for treatment or prevention of autoimmune and inflammatory diseases and conditions by inhibiting or modifying histone demethylation. In a further aspect the invention relates to a method for identifying agents useful in said methods of treatment. The invention particularly describes the role of certain histone demethylase enzymes in these diseases and conditions and their use as therapeutic and screening targets.08-09-2012
20100063134TREATMENT OF NEURODEGENERATIVE DISEASE THROUGH INTRACRANIAL DELIVERY OF SIRNA - The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.03-11-2010
20100035967MODULATION OF TOLL-LIKE RECEPTOR 9 EXPRESSION BY ANTISENSE OLIGONUCLEOTIDES - Antisense oligonucleotide compounds, compositions and methods are provided for down regulating the expression of TLR9. The compositions comprise antisense oligonucleotides targeted to nucleic acids encoding TLR9. The compositions may also comprise antisense oligonucleotides targeted to nucleic acids encoding TLR9 in combination with other therapeutic and/or prophylactic compounds and/or compositions. Methods of using these compounds and compositions for down-regulating TLR9 expression and for prevention or treatment of diseases wherein modulation of TLR9 expression would be beneficial are provided.02-11-2010
20100004315Biodegradable Cross-Linked Branched Poly(Alkylene Imines) - Disclosed is a cross-linked branched poly(alkylenimine) and compositions thereof and nucleotide molecules. Also disclosed are methods for preparing the cross-linked branched poly(alkylenimine).01-07-2010
20110152358Compositions and Methods for Diagnosis and Treatment of Pancreatic Ductal Cancer - The present invention includes compositions and methods for diagnosing and treating pancreatic cancer. These compositions and methods are based on the finding that 14-3-3σ protein is secreted from pancreatic cancer cells and is therefore a specific biomarker protein.06-23-2011
20110152345EBI3, DLX5, NPTX1 AND CDKN3 FOR TARGET GENES OF LUNG CANCER THERAPY AND DIAGNOSIS - The present invention relates to methods for treating or preventing lung cancer by administering a double-stranded molecule against one or more of EBI3, DLX5, NPTX1, CDKN3 or EF-I delta genes or compositions, vectors or cells containing such a double-stranded molecule. The present invention also features methods for diagnosing lung cancer, especially NSCLC or SCLC, using one or more over-expressed genes selected from among EBI3, DLX5, NPTX1, CDKN3 and/or EF-I delta. Also disclosed are methods of identifying compounds for treating and preventing lung cancer, using as an index their effect on the over-expression of one or more of EBI3, DLX5, CDKN3 and/or EF-I delta in the lung cancer, the cell proliferation function of one or more of EBI3, DLX5, NPTX1, CDKN3 and/or EF-I delta or the interaction between CDKN3 and VRS, EF-I beta, EF-I gamma and/or EF-I delta.06-23-2011
20090298913SMALL INTERFERING OLIGONUCLEOTIDES COMPRISING ARABINOSE MODIFIED NUCLEOTIDES - Small interfering ribonucleic acid duplexes that inhibit gene expression containing at least one arabinose modified nucleotide are provided. Preferably, the duplexes contain ribonucleotides at least one arabinose modified nucleotide is 2′-deoxy-2′-fluoroarabinonucleotide (FANA) nucleotide.12-03-2009
20090292004Methods to identify compounds useful for the treatment of proliferative and differentiative disorders - The present invention relates to the discovery and characterization of activity of Fbp1, a substrate-targeting ubiquitin ligase subunit. The invention encompasses interactions between Fbp1 and its substrates, including Fbp5, β-Catenin, and IκBα. The invention also encompasses interactions between the Fbp1 isoform β-Trcp2 and its substrates, including Fbp5, b-Catenin, and IκBα. The present invention relates to screening assays that use Fbp1 and/or β-Trcp2 to identify potential therapeutic agents such as small molecules, compounds or derivatives which modulate Fbp1 and/or β-Trcp2 activity for the treatment of proliferative and differentiative disorders, including infertility, cancer, major opportunistic infections, immune disorders, certain cardiovascular diseases, and inflammatory disorders. The invention also encompasses methods to diagnose and treat Fbp1-related infertility disorders. The invention further encompasses therapeutic protocols and pharmaceutical compositions designed to target Fbp1 and its substrates for the treatment of infertility.11-26-2009
20120202872METHODS AND COMPOSITIONS FOR THE DIAGNOSIS AND PROGNOSIS OF CERVICAL INTRAEPITHELIAL NEOPLASIA AND CERVICAL CANCER - The invention provides methods and compositions for the diagnosis and prognosis of cervical intraepithelial neoplasia and cervical cancer. The methods comprise the step of determining the expression levels or genetic status of specific miRNAs.08-09-2012
20120202871CATIONIC LIPIDS AND METHODS FOR THE DELIVERY OF THERAPEUTIC AGENTS - The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.08-09-2012
20120202870TREATMENT OF INFLAMMATORY DISEASES USING MIR-124 - Methods of treating, reducing the risk of developing, or delaying the onset of an inflammatory disease are disclosed. The methods involved providing a subject with or at risk of developing an inflammatory disease and administering to the subject an effective amount of a first therapeutic composition comprising miR-124. Further provided are methods of diagnosing a subject with or at risk of developing an inflammatory disease.08-09-2012
20110152349COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF IL-18 GENES - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a IL-18 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a IL-18 gene using said pharmaceutical composition; and methods for inhibiting the expression of IL-18 in a cell.06-23-2011
20090221678Compositions and Methods for Treatment of Prostate and Other Cancers - Therapeutic agents which target heat shock protein (hsp) 27 in vivo are used to provide treatment to individuals, particularly human individuals, suffering from prostate cancer and other cancers that overexpress hsp27. A therapeutic agent, for example an antisense oligonucleotide or RNAi nucleotide inhibitor with sequence specificity for hsp27 mRNA, for example human hsp27 mRNA, is administered to an individual suffering from prostate cancer or some other cancer expressing elevated levels of hsp 27 in a therapeutically effective amount. The therapeutic agent is suitably formulated into a pharmaceutical composition which includes a pharmaceutically acceptable carrier, and packaged in dosage unit form. A preferred dosage unit form is an injectable dosage unit form.09-03-2009
20120122956METHODS FOR INHIBITING ANGIOGENESIS WITH MULTI-ARM POLYMERIC CONJUGATES OF 7-ETHYL-10-HYDROXYCAMPTOTHECIN - The present invention relates to methods of inhibiting angiogenesis in mammals. The present invention includes administering polymeric prodrugs of 7-ethyl-10-hydroxycamptothecin to the mammals in need thereof. The present invention also relates to methods of treating a disease associated with angiogenesis in mammals by administering polymeric prodrugs of 7-ethyl-10-hydroxycamptothecin to the mammals in need thereof.05-17-2012
20090209621Compositions and methods for decreasing microrna expression for the treatment of neoplasia - The invention generally features compositions and methods that are useful for treating or diagnosing a neoplasia. The invention is based in part on the observation that c-Myc activated expression of a cluster of six miRNAs on human chromosome 13. Accordingly, the invention provides therapeutic compositions and methods for altering the expression of a microRNA of the invention thereby treating a neoplasia, as well as compositions and methods for diagnosing a neoplasia.08-20-2009
20110178155SILENCING OF CSN5 GENE EXPRESSION USING INTERFERING RNA - The present invention provides compositions comprising nucleic acids that target CSN5 gene expression and methods of using such compositions to silence CSN5 gene expression. More particularly, the present invention provides unmodified and chemically modified interfering RNA molecules which silence CSN5 gene expression and methods of use thereof, e.g., for treating cell proliferative disorders such as cancer. The present invention also provides nucleic acid-lipid particles that target CSN5 gene expression comprising an interfering RNA molecule, a cationic lipid, a non-cationic lipid, and optionally a conjugated lipid that inhibits aggregation of particles.07-21-2011
20120277292ANTISENSE OLIGONUCLEOTIDES AGAINST cPLA2, COMPOSITIONS AND USES THEREOF - Antisense oligonucleotides against cPLA11-01-2012
20120277291PREVENTIVE FOR ADHESION FOLLOWING ABDOMINAL SURGERY - The present inventors discovered that oligonucleotides which suppress midkine expression and antibodies which suppress midkine activity can be used to prevent post-surgical intraperitoneal adhesions.11-01-2012
20120277290TREATMENT OF SEX HORMONE BINDING GLOBULIN (SHBG) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO SHBG - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Sex Hormone Binding Globulin (SHBG), in particular, by targeting natural antisense polynucleotides of Sex Hormone Binding Globulin (SHBG). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of SHBG.11-01-2012
20120277288Means and Methods for the Specific Modulation of Target Genes in the CNS and the Eye and Methods for Their Identification - Provided are methods for the treatment of disorders of the central nervous system (CNS) and the eye. In particular, use of compositions comprising a compound capable of modulating a target gene or gene product is described for the preparation of a pharmaceutical composition for the treatment of disorders of the CNS and/or the eye, wherein the composition is designed to be administered outside the blood-CNS and the blood-retina barriers. Furthermore, methods are provided for identifying and obtaining nucleic acid molecules encoding polypeptides involved in CNS disorders or of the eye, methods for diagnosing said disorders as well as transgenic animal deficient in the expression of target genes identified in accordance with the described method. In addition, methods of identifying and isolating drugs that are particularly useful for the treatment of disorders related to the CNS and/or the eye are disclosed.11-01-2012
20120277289ACTIVITY GENERATING DELIVERY MOLECULES - Activity-generating delivery molecules comprising the structure R11-01-2012
20100280098RECEPTOR TARGETED OLIGONUCLEOTIDES - Disclosed herein are oligonucleotide conjugates that include ligands that target cell receptors that mediate endocytosis. The ligands can include peptides and small molecules. The conjugates can include carrier macromolecules to which the ligands and oligonucleotides are attached, or conjugates where an oligonucleotide is attached to a ligand in the absence of a carrier macromolecule. The oligonucleotides can include therapeutic oligonucleotides, such as siRNA, antisense RNA and miRNA. The ligand can be an RGD peptide. Also disclosed herein are methods of delivering the conjugates to cells in subjects.11-04-2010
20090239936Prophylactic and Therapeutic Agent for Cancer - A Ras, Raf, MEK, ERK or RSK inhibitor, namely a P-glycoprotein expression inhibitor or a BCRP expression inhibitor, can be screened by utilizing the MAPK signaling activity as an indicator. It becomes possible to provide an anticancer agent which is reduced in resistance acquisition, and also provide an agent for preventing the resistance against an anticancer agent, which can enhance the therapeutic effect of the anticancer agent against cancer.09-24-2009
20110009466METHODS OF INCREASING GENE EXPRESSION THROUGH RNA PROTECTION - This invention relates to the use of one or more RNA target protectors to inhibit the binding of an RNA, e.g., small RNA, to a target RNA (e.g., a target mRNA), thus increasing the stability of the target RNA and its function (e.g., increasing the gene expression of the gene corresponding to a target mRNA). An RNA target protector may be, for example, an oligonucleotide, e.g., a morpholino, or a small molecule. The invention further relates to the treatment of a human patient in need thereof with one or more RNA target protectors.01-13-2011
20110166201MIRNAS AS THERAPEUTIC TARGETS IN CANCER - MicroRNAs (miRNAs) are a class of non-coding small RNA molecules that regulate gene expression at the post-transcriptional level by interacting with 3′ untranslated regions (UTRs) of their target mRNAs. The invention relates to the application of miR-192 and miR-215. Both of these miRNAs impact cellular proliferation through the p53-miRNA circuit, and interact with dihydrofolate reductase (DHFR) and thymidylate synthase (TS). Particularly, upregulation of these miRNAs reduces cellular proliferation. The invention relates to this discovery. For example, inhibiting miR-192 and/or miR-215 sensitizes a neoplasm or a subject with a neoplasm to chemotherapeutic agents. Furthermore, measuring the levels of miR-192 and/or miR-215 provides one with information regarding whether the neoplasm or subject will respond to chemotherapeutic agents. Accordingly, the invention relates to composition and methods relating to the identification, characterization and modulation of the expression of miR-192 and miR-215.07-07-2011
20110021601COMPOSITION CONTAINING MICRORNA-21 INHIBITOR FOR ENHANCING RADIATION SENSITIVITY - Disclosed is a radiation sensitivity-enhancing composition in which a microRNA-21 inhibitor acts as an active ingredient. The microRNA-21 inhibitor is an antisense nucleic acid molecule binding complementarily to microRNA-21. The composition can be administered to a patient in conjunction with irradiation. The inhibitor can act as a radiosensitizer, enhancing the therapeutic effect of such irradiation on cancer high in microRNA-21 expression level, particularly, glioma.01-27-2011
20110054009MicroRNA-Based Methods and Compositions for the Diagnosis, Prognosis and Treatment of Prostate Related Disorders - Methods and compositions for the diagnosis, prognosis and/or treatment of prostate associated disorders are disclosed.03-03-2011
20110054008RNAi Inhibition of Serum Amyloid A For Treatment of Glaucoma - RNA interference is provided for inhibition of serum amyloid A mRNA expression in glaucomas involving SAA expression.03-03-2011
20110054007COMPOSITIONS AND METHODS TO CONTROL INSECT PESTS - Methods and compositions are provided which employ a silencing element that, when ingested by a pest, such as a Coleopteran plant pest or a 03-03-2011
20110054005Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same - The present invention provides a polynucleotide that not only has a high RNA interference effect on its target gene, but also has a very small risk of causing RNA interference against a gene unrelated to the target gene. A sequence segment conforming to the following rules (a) to (d) is searched from the base sequences of a target gene for RNA interference and, based on the search results, a polynucleotide capable of causing RNAi is designed, synthesized, etc.: 03-03-2011
20110054004METHOD FOR REDUCING SCARRING DURING WOUND HEALING USING ANTISENSE COMPOUNDS DIRECTED TO CTGF - This invention provides a method for reducing hypertropic scarring resulting from dermal wound healing in a subject in need which comprises administering to the subject an antisense oligonucleotide which inhibits expression of connective tissue growth factor (CTGF) in an amount effective to inhibit expression of CTGF and thereby reduce hypertrophic scarring.03-03-2011
20100286241COMPOSITIONS COMPRISING K-RAS SIRNA AND METHODS OF USE - The present invention provides nucleic acid molecules that inhibit K-ras expression. Methods of using the nucleic acid molecules are also provided.11-11-2010
20100286242siRNA-Mediated Gene Silencing of Synuclein - The present invention is directed to small interfering RNAs that down regulate expression of a synuclein gene and methods of using the small interfering RNAs.11-11-2010
20100286238SUPPRESSION OF VIRUSES INVOLVED IN RESPIRATORY INFECTION OR DISEASE - The present invention concerns methods and reagents useful in decreasing the level of or severity of respiratory infection or disease due to paramyxoviruses, such as RSV or HPIV, or coronavirus infection. Particularly, the invention relates to modulating gene expression using a multitargeting interfering RNA molecules that target multiple target sites on one or more pre-selected RNA molecules.11-11-2010
20100286246IDENTIFICATION OF NOVEL GENES CODING FOR SMALL TEMPORAL RNAS11-11-2010
20100286239ANTISENSE OLIGONUCLEOTIDES FOR TREATING ATOPIC DISEASES AND NEOPLASTIC CELL PROLIFERATION - The present invention relates to the use of antisense oligonucleotides directed against specific nucleic acid sequences coding for receptors, alone or in combination, in order to inhibit the inflammatory reaction that is present in asthma, atopy or hypereosinophilia and to inhibit neoplastic cell proliferation. The antisense oligonucleotides of the present invention are used for treating and/or preventing asthma, allergy, hypereosinophilia, general inflammation or cancer. The oligonucleotides of the present invention are more specifically directed against nucleic acid sequences coding for a CCR3 receptor, a common sub-unit of IL-4 and IL-13 receptors, or a common sub-unit of IL-3, IL-5 and GM-CSF receptors.11-11-2010
20100286245IDENTIFICATION OF NOVEL GENES CODING FOR SMALL TEMPORAL RNAS11-11-2010
20100286243MIG-7 AS A SPECIFIC ANTICANCER TARGET - Aspects of the present invention provide novel Mig-7 encoding nucleic acids and Mig-7 polypeptides, RNAi (such as siRNA), recombinant DNA expression systems and host cells containing same, as well as methods of inhibiting expression of the subject nucleic acid molecules, inhibiting production of the encoded proteins or polypeptides, inhibiting metastasis of a carcinoma cell in a subject (including in humans), inhibiting migration/invasion of and mimicking of normal cells by carcinoma cells in a subject, detecting the presence of a cancer cell (e.g., a migrating/invading cancer cell or carcinoma cell mimic, tumor neovas-cularization, and/or vascular mimicry) in a sample of a subject's tissue or body fluids, and inhibiting the migration/invasion of an endothelial cell mimicking by a placental cell into the blood stream or vessels of a female mammal. Particular aspects relate to novel anti-Mig-7 antibodies, diagnostic and/or prognostic methods, and therapeutic methods comprising use of the inventive nucleic acids, polypeptides and antibodies or derivatives thereof.11-11-2010
20100286240MicroRNAs FOR INHIBITING VIRAL REPLICATION - The present invention relates to reducing accumulation of viral genomes in a target cell. In particular the present invention provides compositions and methods for combating viral infection through RNA interference. Specifically the present invention provides cellular microRNA mimics for treating virus-infected subjects.11-11-2010
20100286236Dosage of oligonucleotides suitable for the treatment of tumors - A method for preventing and/or treating a tumor, the method comprising: intravenously administering an antisense oligonucleotide in an amount of between about 400 to about 800 mg/m11-11-2010
20100286234Pharmaceutical Composition Comprising Anti-Mirna Antisense Oligonucleotides - The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 26 nucleobases which are complementary to human microRNAs selected from the group consisting of miR19b, miR21, miR122a, miR155 and miR375. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogues into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC.11-11-2010
20100286229Modulation of Androgen Receptor for Treatment of Prostate Cancer - The present invention provides methods for the reduction of endotoxins in a plasmid preparation using a carbohydrate non-ionic detergent with silica chromatography.11-11-2010
20110112167THERAPEUTIC AGENTS AND TARGETS - The present invention relates to diagnostic and therapeutic methods in relation to diabetic complications, such as blindness, nephropathy and cardiovascular disease, and inflammatory conditions, such as angina, arthritis, empyema pharyngitis and urinary tract infection. Diagnostic methods involve screening for up regulated expression of decor (Den) or thioredoxin-like protein 19 (TLP 19). Therapeutic methods involve modulation of expression or activity o Den or TLP 19. The invention also relates to screening methods for identifying functional signal sequences to screen for secreted, membrane-bound and exported proteins and cell surface receptors.05-12-2011
20110136889COMPOSITIONS AND THEIR USES DIRECTED TO ACEYTL-COA CARBOXYLASES - Disclosed herein are compounds, compositions and methods for modulating the expression of ACC1 or ACC2 or both in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.06-09-2011
20110263675METHODS OF MODULATING MESENCHYMAL STEM CELL DIFFERENTIATION - The present disclosure includes compositions and methods for modulating the differentiation of cells having osteogenic differentiation potential (such as mesenchymal stem cells (MSCs)) towards the osteogenic fate, and for obtaining diagnostic and prognostic information relating to diseases and disorders characterized by defects in osteogenic differentiation. The compositions include miRNAs, rm′RNA mimics, miRNA inhibitors, and siRNAs.10-27-2011
20120302626MICRORNA AND USE THEREOF IN IDENTIFICATION OF B CELL MALIGNANCIES - Disclosed are nucleic acid sequences, including microRNA sequences and cDNA sequences, as well as vectors, DNA libraries, microarrays, and recombinant cells comprising the nucleic acid sequences described herein. Methods of determining the B cell stage from which a B cell malignancy is derived. Methods of identifying B cell malignancies are also provided. Methods of diagnosing B cell malignancies are provided. Such methods comprise, in certain embodiments, detecting one or more microRNAs or cDNAs as disclosed herein.11-29-2012
20120302625SUPERCOILED MINICIRCLE DNA FOR GENE THERAPY APPLICATIONS - The present invention relates to nucleic acid molecule compositions comprising minivectors encoding a nucleic acid sequence and methods of gene therapy using minivectors encoding a nucleic acid sequence.11-29-2012
20120302624BIOMARKER FOR IDENTIFYING SUBGROUP OF EARLY-STAGE LUNG ADENOCARCINOMA PATIENTS - The preset invention relates to a biomarker for identifying the subgroup of early-stage lung adenocarcinoma patients in early-stage non-small cell lung cancer (NSCLC), which is T-lymphokine-activated killer cell-originated protein kinase (TOPK), and a therapeutic target for lung cancer.11-29-2012
20120302623Novel Activation and Transfer Cascade for Ubiquitin - A novel activating enzyme for ubiquitin, Uba6, is provided. Compositions and methods for inhibiting ubiquitin via the Uba6 pathway are provided. Methods of identifying novel inhibitors of ubiquitination are also provided. Novel RNAi molecules are also provided.11-29-2012
20120302622CONJUGATES, PARTICLES, COMPOSITIONS, AND RELATED METHODS - Particles and conjugates for delivering nucleic acid agents. Compositions containing the particles, the conjugates, or both. Methods of using the particles, the conjugates, and the compositions.11-29-2012
20110257248RNAi-RELATED INHIBITION OF TNFa SIGNALING PATHWAY FOR TREATMENT OF OCULAR ANGIOGENESIS - RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enzyme (TACE/ADAM17) mRNA expression. Silencing such TNFα targets, in particular, is useful for treating patients having a TNFα-related condition or at risk of developing a TNFα-related condition, such as ocular angiogenesis, retinal ischemia, and diabetic retinopathy.10-20-2011
20110257247Method of Treating Neurodegenerative Disease - Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.10-20-2011
20110257246RNAi-MEDIATED INHIBITION OF HIF1A FOR TREATMENT OF OCULAR ANGIOGENESIS - RNA interference is provided for inhibition of HIF1A mRNA expression for treating patients with ocular angiogenesis, particularly for treating retinal edema, diabetic retinopathy, sequela associated with retinal ischemia, posterior segment neovascularization (PSNV), and neovascular glaucoma, and for treating patients at risk of developing such conditions.10-20-2011
20110118332VIRAL MICRORNA - The present invention relates, in general, to micro RN As and. in particular, to viral microRNAs expressed by Herpes Simplex Vims 1 (HSV-1) or Herpes Simplex Virus 2 (HSV-2), to agents that inhibit such microRNAs and to methods of treatment based on the use of such agents.05-19-2011
20110263684Lipid Formulated Compositions and Methods for Inhibiting Expression of Serum Amyloid A Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA.10-27-2011
20110263685Method for Identification of Sensitivity of a Patient to Telomerase Inhibition Therapy - The invention provides methods for determining the susceptibility of cancer patients to developing adverse reactions if treated with a telomerase inhibitor drug by measurement of telomere length in appropriate cells of the patient prior to initiation of the telomerase inhibitor treatment.10-27-2011
20110263679C12ORF48 AS A TARGET GENE FOR CANCER THERAPY AND DIAGNOSIS - Objective methods for diagnosing a predisposition to developing pancreatic cancer and prostate cancer, particularly pancreatic ductal adenocarcinoma (PDAC) and castration-resistant prostate cancer, are described herein. In one embodiment, the diagnostic method involves the step of determining an expression level of C12ORF48 using siRNAs targeting the C12ORF48 gene. The invention also features products such as siRNAs as well as to compositions containing them. The present invention further provides methods of screening for therapeutic agents useful in the treatment of C12ORF48 associated disease, such as a cancer, e.g. pancreatic cancer and prostate cancer, as well as methods of inhibiting the cell growth and treating or alleviating one or more disease symptoms. The invention also features products such as double stranded molecules, as well as vectors and compositions containing them.10-27-2011
20110263682METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53 - The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.10-27-2011
20110263681Organic Compositions to Treat Beta-ENaC-Related Diseases - The present disclosure relates to RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.10-27-2011
20100240730RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, cosmetic, cosmeceutical, prophylactic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of disease (e.g., cancer, proliferative, inflammatory, metabolic, autoimmune, neurologic, ocular diseases), condition, trait (e.g., hair growth and removal), genotype and phenotype that responds to modulation of gene expression or activity in a cell, tissue, or organism. Such small nucleic acid molecules can be administered systemically, locally, or topically.09-23-2010
20100298412Disulfide Chemotherapeutic Agents and Methods of Use Thereof - Compositions and methods for the treatment of cancer are provided.11-25-2010
20100298408Oligonucleotide Compositions with Enhanced Efficiency - The oligonucleotide compositions of the present invention make use of combinations of oligonucleotides. In one aspect, the invention features an oligonucleotide composition including at least 2 different oligonucleotides targeted to a target gene. This invention also provides methods of inhibiting protein synthesis in a cell and methods of identifying oligonucleotide compositions that inhibit synthesis of a protein in a cell.11-25-2010
20100298409COMPOSITIONS COMPRISING STAT3 SIRNA AND METHODS OF USE THEREOF - The present invention provides nucleic acid molecules that inhibit STAT3 expression. Methods of using the nucleic acid molecules are also provided.11-25-2010
20100298404Translocation and mutant ROS kinase in human non-small cell lung carcinoma - In accordance with the invention, a novel gene translocation, (4p15, 6q22), in human non-small cell lung carcinoma (NSCLC) that results in a fusion proteins combining part of Sodium-dependent Phosphate Transporter Isoform NaPi-3h protein (SLC34A2) with Proto-oncogene Tyrosine Protein Kinase ROS Precursor (ROS) kinase has now been identified. The SLC34A2-ROS fusion protein is anticipated to drive the proliferation and survival of a subgroup of NSCLC tumors. The invention therefore provides, in part, isolated polynucleotides and vectors encoding the disclosed mutant ROS kinase polypeptides, probes for detecting it, isolated mutant polypeptides, recombinant polypeptides, and reagents for detecting the fusion and truncated polypeptides. The disclosed identification of the new fusion protein enables new methods for determining the presence of these mutant ROS kinase polypeptides in a biological sample, methods for screening for compounds that inhibit the proteins, and methods for inhibiting the progression of a cancer characterized by the mutant polynucleotides or polypeptides, which are also provided by the invention.11-25-2010
20110136892Targeting TGF-beta as a Therapy for Alzheimer's Disease - The invention includes compositions and methods for enhancing peripheral macrophage Aβ phagocytosis activity. The invention includes inhibiting a component of TGF-β signaling pathway in peripheral macrophages to promote central nervous system infiltration and beneficial cerebral Aβ clearance. Inhibition of TGF-β signaling in peripheral macrophages represents an advantageous anti-amyloid therapeutic approach for Alzheimer's disease.06-09-2011
20100305191RNA INTERFERENCE MEDIATED INHIBITION OF ADENOSINE A1 RECEPTOR (ADORA1) GENE EXPRESSION USING SHORT INTERFERING RNA - The present invention concerns methods and reagents useful in modulating adenosine A1 receptor (ADORA1) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small interfering RNA (siRNA) molecules capable of mediating RNA interference (RNAi) against ADORA1 and related receptors.12-02-2010
20100305193RNAI-MEDIATED INHIBITION OF GREMLIN FOR TREATMENT OF IOP-RELATED CONDITIONS - RNA interference is provided for inhibition of gremlin in intraocular pressure-related conditions, including ocular hypertension and glaucoma such as normal tension glaucoma and open angle glaucoma.12-02-2010
20100305192COMPOSITIONS AND METHODS FOR SHORT INTERFERING NUCLEIC ACID INHIBITION OF NAV1.8 - The invention provides short interfering nucleic acids, either single-stranded or double-stranded, that cause RNAi-induced degradation of mRNA from the Na12-02-2010
20100305190NUCLEIC ACID COMPLEX AND NUCLEIC ACID DELIVERY COMPOSITION - The present invention provides a nucleic acid complex with low toxicity and high safety that can persistently maintain a nucleic acid, such as siRNA or the like, in a cell; and a nucleic acid delivery composition that can efficiently deliver the nucleic acid complex into a cell. A nucleic acid complex with low toxicity and high safety that can persistently maintain a nucleic acid in a cell can be obtained by forming a complex using a nucleic acid to be introduced into a cell, and a highly branched cyclic dextrin. Moreover, when a carrier comprising (A) a diacylphosphatidylcholine, (B) cholesterol and/or a derivative thereof, and (C) an aliphatic primary amine is used as a nucleic acid delivery carrier to introduce the nucleic acid complex into a cell, the safety, the efficiency of intracellular delivery, and the persistence of the nucleic acid in the cell can be further improved.12-02-2010
20100305189PHARMACEUTICAL COMPOSITION FOR PREVENTING, STABILISING AND/OR INHIBITING BLOOD AND LYMPH VASCULARIZATION - A pharmaceutical composition including as active agent, an antisens oligonucleotide having the sequence SEQ ID NO: 1 in a concentration from about 0.40 mg/ml to about 2 mg/ml and the use thereof for preventing, stabilizing and/or inhibiting blood and lymph vascularization.12-02-2010
20100210708COMPOUND PROFILING METHOD - The present invention provides a method for deriving an upstream or downstream component of a component necessary for phenotypic alteration of a living organism, the method comprising the steps of: specifying a pathway of interest related to the phenotypic alteration and a reference pathway different from the pathway of interest, and specifying a stimulant of interest and a reference stimulant which respectively stimulate the pathway of interest and the reference pathway; giving the stimulant of interest to the living organism to identify a collection of components of interest necessary for the phenotypic alteration; giving the reference stimulant to the living organism to identify a collection of reference components necessary for the phenotypic alteration; calculating an intersection between the collections of the components of interest and the reference components; and calculating a differential collection by subtracting the intersection from the collection of components of interest.08-19-2010
20100324114Methods and kits to detect hereditary angioedema type III - The present invention relates to a method of diagnosing hereditary angioedema type III (HAE III) or a predisposition thereto in a subject being suspected of having developed or of having a predisposition to develop a hereditary angioedema type III or in a subject being suspected of being a carrier for hereditary angioedema type III, the method comprising determining in vitro from a biological sample of said subject the presence or absence of a disease-associated mutation in a nucleic acid molecule regulating the expression of or encoding coagulation factor XII; wherein the presence of such a mutation is indicative of a hereditary angioedema type III or a predisposition thereto. The present invention also relates to a method of diagnosing hereditary angioedema type III (HAE III) or a predisposition thereto in a subject being suspected of having developed or of having a predisposition to develop a hereditary angioedema type III or in a subject being suspected of being a carrier for hereditary angioedema type III, the method comprising assessing the presence, amount and/or activity of coagulation factor XII in said subject and including the steps of: (a) determining from a biological sample of said subject in vitro, the presence, amount and for activity of: (i) a (poly)peptide encoded by the coagulation factor XII gene; (ii) a substrate of the (poly)peptide of (i); or (iii) a (poly)peptide processed by the substrate mentioned in (ii); (b) comparing said presence, amount and/or activity with that determined from a reference sample; and (c) diagnosing, based on the difference between the samples compared in step (b), the pathological condition of a hereditary angioedema type III or a predisposition thereto. The present invention also relates to a method of identifying a compound modulating coagulation factor XII activity which is suitable as a medicament or a lead compound for a medicament for the treatment and/or prevention of hereditary angioedema type III, the method comprising the steps of: (a) in vitro contacting a coagulation factor XII (poly)peptide or a functionally related (poly)peptide with the potential modulator; and (b) testing for modulation of coagulation factor XII activity, wherein modulation of coagulation factor XII activity is indicative of a compound's suitability as a medicament for the treatment and/or prevention of hereditary angioedema type III. Furthermore, the present invention relates to gene therapy methods and to a kit for diagnosing hereditary angioedema type III.12-23-2010
20100324115Treatment of Squamous Cell Carcinoma with HSP27 Antisense Oligonucleotides and Radiotherapy - Squamous cell carcinomas, such as squamous head and neck cancer, are treated with a combination of radio-therapy and a therapeutic agent that reduces the amount of hsp27 in the squamous cancer cells. In specific embodiments, the therapeutic agent that reduces the amount of hsp27 is an antisense oligonucleotide therapeutic agent.12-23-2010
20100324116FAS/FASL OR OTHER DEATH RECEPTOR TARGETED METHODS AND COMPOSITIONS FOR KILLING TUMOR CELLS - Provided herein are methods and compositions for killing tumor cells. In certain embodiments, compositions can promote apoptosis by down-regulating FAPP2 and PATZ1 products.12-23-2010
20100324113Delivery Method - The present invention relates, in general, to siRNA and, in particular, to a method of effecting targeted delivery of siRNAs and to compounds suitable for use in such a method.12-23-2010
20100324119REDUCING IRF4, DUSP22, OR FLJ43663 POLYPEPTIDE EXPRESSION - This document relates to the activity of interferon regulatory factor 4 (IRF4) in T-cell lymphomas. For example, methods and materials involved in reducing the expression of an IRF4 polypeptide in T-cell lymphoma cells and identifying agents having the ability to reduce expression of an IRF4 polypeptide in T-cell lymphoma cells are provided. This document also relates to reducing DUSP22 or FLJ43663 polypeptide activity in T-cell lymphomas. For example, methods and materials involved in reducing the expression of DUSP22 polypeptides and/or FLJ43663 polypeptides in T-cell lymphoma cells and identifying agents having the ability to reduce expression of DUSP22 polypeptides and/or FLJ43663 polypeptides in T-cell lymphoma cells are provided.12-23-2010
20100168205Methods and Compositions for Prevention or Treatment of RSV Infection Using Modified Duplex RNA Molecules - Methods and compositions are provided for the prevention or treatment of RSV infection in a human. The methods include administering one or more doses of a composition comprising an siRNA. The dose can be formulated for topical or parenteral administration. Topical administration includes administration as a nasal spray, or by inhalation of respirable particles or droplets. The siRNA preferably comprises a sense strand and antisense strand with modified nucleotides.07-01-2010
20100179212Pharmaceutical composition which improves in vivo gene transfer - A pharmaceutical composition which combines a tetrafunctional copolymer with a nucleic acid, said copolymer having formula I (namely a poloxamine), and preferably taking the form of one of the cationic mineral or organic salts thereof. The composition can be used to improve in vivo gene transfer.07-15-2010
20110257244CHEMICALLY MODIFIED OLIGONUCLEOTIDES AND SMALL MOLECULES FOR USE IN REDUCING MICRO RNA ACTIVITY LEVELS AND USES THEREOF - This invention relates generally to chemically modified oligonucleotides useful for modulating activity of microRNAs and pre-microRNAs. More particularly, the invention relates to single stranded chemically modified oligonucleotides for inhibiting microRNA and pre-microRNA activity and to methods of making and using the modified oligonucleotides.10-20-2011
20100173971Genetic changes in ATM and ATR/CHEK1 as prognostic indicators in cancer - The present invention relates to the discovery that, in human cancer, an 1107-08-2010
20100168206GNAQ Targeted dsRNA Compositions And Methods For Inhibiting Expression - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.07-01-2010
20110065778RNA Interference Mediated Inhibition of Muscarinic Colinergic Receptor Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods useful for modulating the expression of genes associated with respiratory and pulmonary disease, such as cholinergic muscarinic receptor genes, using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of cholinergic muscarinic receptor genes, or other genes involved in pathways of cholinergic muscarinic receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of M3 muscarinic acetylcholine receptor or cholinergic receptor muscarinic 3 (CHRM3).03-17-2011
20110190379PLANT PATHOGEN RESISTANCE - The present invention relates to a method for protecting a plant from infection by a pathogen by decreasing the presence of a plant hormone or reducing the responsiveness of a plant to a plant hormone. In particular, the invention related to infection by a necrotrophic pathogen, such as 08-04-2011
20100305187METHODS AND COMPOUNDS FOR TREATING DISEASES CAUSED BY REACTIVE OXYGEN SPECIES - Provided is a method of treating a patient having an inflammatory disease by using a compound which inhibits the complex I-mediated ROS production, a medicament containing such compound and methods for screening for such compounds.12-02-2010
20100190838Connective Tissue Growth Factor Fragments and Methods and Uses Thereof - The present invention is directed to CTGF fragments comprising at least exon 2 or exon 3 of CTGF and having the ability to induce extracellular matrix synthesis, in particular, collagen synthesis and myofibroblast differentiation. The present invention is further directed to methods using said CTGF fragments to identify compositions which modulate the activity of said CTGF fragments and to the compositions so identified. The invention also relates to methods of treating CTGF-associated disorders and diseases associated with the overproduction of the extracellular matrix.07-29-2010
20110190382Treatment of Cancer by Inhibition of IGFBPs and Clusterin - Agents that reduce the amount of IGFBP-2 and/or IGFBP-5 and that are known to be useful in the treatment of cancer result in increased expression of the protein clusterin. Since clusterin can provide protection against apoptosis, this secondary effect detracts from the efficacy of the therapeutic agent. In overcoming this, the present invention provides a combination of therapeutic agents that is useful in the treatment of cancer. The combination includes an agent that reduces the amount of IGFBP-2 and/or IGFBP-5 and that stimulates expression of clusterin as a secondary effect, and an oligonucleotide that is effective to reduce the amount of clusterin in cancer cells. In some embodiments of the invention, the agent that reduces IGFBP-2 and/or IGFBP-5 is a bispecific antisense species. The oligonucleotide may be an antisense oligonucleotide or an RNAi oligonucleotide.08-04-2011
20110190381RNAI-MEDIATED INHIBITION OF FRIZZLED RELATED PROTEIN-1 FOR TREATMENT OF GLAUCOMA - RNA interference is provided for inhibition of Frizzled Related Protein-1 mRNA expression, in particular, for treating patients having glaucoma or at risk of developing glaucoma.08-04-2011
20110190380METHODS FOR DELIVERY OF SIRNA TO BONE MARROW CELLS AND USES THEREOF - The present invention relates to a method for the delivery of therapeutic oligonucleotides to bone marrow, and in particular delivery of siRNA to a subset of bone marrow cells. The method comprises systemically administering siRNA to a subject in need thereof, to reduce or inhibit expression of a gene associated with a disease or disorder or to symptoms associated with a disease or disorder associated with the cells. The invention further relates to chemically modified siRNA compounds, to pharmaceutical compositions comprising such compounds and to methods of using such compounds and compositions in the treatment of disease.08-04-2011
20110190378MIR 204, MIR 211, THEIR ANTI-MIRS, AND THERAPEUTIC USES OF SAME - Embodiments of the invention provide methods of preventing or treating detrimental epithelial cell proliferation, loss of epithelial cell differentiation, age-related macular degeneration and/or proliferative vitreal retinopathy in an individual comprising administering to an individual in need thereof an effective amount of miR 204, an effective amount of miR 211, or an effective amount of a mixture of miR 204 and miR 211. A further embodiment of the invention provides a method of facilitating the transport of a substance across an epithelium in an individual comprising administrating to an individual an effective amount of anti-miR 204, an effective amount of anti-miR 211, or an effective amount of a mixture of anti-miR 204 and anti-miR 211. Additional embodiments of the invention include pharmaceutical compositions of miR 204 and/or miR 211 and pharmaceutical compositions of anti-miR 204 and/or anti-miR 211.08-04-2011
20110190376RNAi-RELATED INHIBITION OF TNFa SIGNALING PATHWAY FOR TREATMENT OF MACULAR EDEMA - RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enzyme (TACE/ADAM17) mRNA expression. Silencing such TNFα targets, in particular, is useful for treating patients having or at risk of developing macular edema.08-04-2011
20110190375COMPOSITIONS COMPRISING CMYC SIRNA AND METHODS OF USE THEREOF - The present invention provides nucleic acid molecules that inhibit c-Myc expression. Methods of using the nucleic acid molecules are also provided.08-04-2011
20110190374METHODS OF TREATING A MEIOTIC KINESIN ASSOCIATED DISEASE - The invention provides methods of treating a meiotic kinase-associated disease, preferably the meiotic kinase HSET, by administering an inhibitor of the meiotic kinase. Preferably, the disease is associated with the presence of supernumerary centrosomes, such as cancer. Methods of inhibiting the growth of a tumor cell by contacting the cell with an inhibitor of a meiotic kinase, preferably HSET, are also provided. Screening methods for identifying inhibitors of the meiotic kinase HSET are also provided. Methods of selecting subjects for treatment with an inhibitor of a meiotic kinase, such as HSET, are also provided.08-04-2011
20110190373METHODS AND COMPOSITIONS FOR THE TREATMENT OR PREVENTION OF PATHOLOGICAL CARDIAC REMODELING AND HEART FAILURE - The invention relates to methods of treating or preventing pathological cardiac remodeling and/or preventing heart failure. These methods include the administration of a PDE1 inhibitor to a patient under conditions effective to treat or prevent pathological cardiac remodeling, and therefore heart failure that occurs as a result of such remodeling. Pharmaceutical compositions and delivery vehicles that can be used in the methods of the present invention are also disclosed herein.08-04-2011
20110190372COMPOSITIONS AND METHODS FOR TREATING INFLAMMATORY DISORDERS - Compositions and methods for antagonizing miRNAs that are overexpressed in chronic, non-healing wounds, as compared to healthy tissue, are disclosed. The miRNA antagonists are oligonucleotides that hybridize to selected pre-miRNA or mature miRNAs and prevent the miRNAs from binding to and downregulating their target mRNAs. Methods of using the miRNA antagonists to treat inflammatory disorders, including to promote healing of chronic, non-healing wounds and acute wounds are provided.08-04-2011
20110190371GENETIC SUPPRESSION AND REPLACEMENT - Methods and agents for suppressing expression of a mutant allele of a gene and providing a replacement nucleic acid are provided. The methods of the invention provide suppression effectors such as, for example, antisense nucleic acids, ribozymes, or RNAi, that bind to the gene or its RNA. The invention further provides for the introduction of a replacement nucleic acid with modified sequences such that the replacement nucleic acid is protected from suppression by the suppression effector. The replacement nucleic acid is modified at degenerate wobble positions in the target region of the suppression effector and thereby is not suppressed by the suppression effector. In addition, by altering wobble positions, the replacement nucleic acid can still encode a wild type gene product. The invention has the advantage that the same suppression strategy could be used to suppress, in principle, many mutations in a gene. Also disclosed is a transgenic mouse that expresses human rhodopsin (modified replacement gene) and a transgenic mouse that expresses a suppression effector targeting rhodopsin. Also disclosed in intraocular administration of siRNA.08-04-2011
20110190370MODULATION OF HIF1(ALPHA) AND HIF2(ALPHA) EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of HIF1α and/or HIF2α. The compositions comprise oligonucleotides, targeted to nucleic acid encoding HIF1α and HIF2α. Methods of using these compounds for modulation of HIF1α and/or HIF2α expression and for diagnosis and treatment of disease associated with expression of HIF1α and/or HIF2α are provided.08-04-2011
20110152352SMAD PROTEINS CONTROL DROSHA-MEDIATED MIRNA MATURATION - The invention, in some aspects, relates to compositions and methods useful for modulating expression of miRNAs that are regulated by the TGF-β/BMP signaling pathway. In some aspects, the invention relates, to oligonucleotides comprising a CAGRN-motif that modulate expression of miRNAs that are regulated by TGF-β/BMP signaling pathway. The invention, in some aspects, relates to composition and methods useful for inhibiting microRNA processing. In some aspects, the invention relates to composition and methods for treating TGF-Beta/BMP mediated disorders.06-23-2011
20110178159INHIBITORY RNAS THAT REGULATE HEMATOPOIETIC CELLS - Provided are compositions and methods for preventing, treating, ameliorating or diagnosing conditions or diseases involving a myeloid cell proliferation disorder. Such compositions and methods target miRNA function myeloproliferative diseases. More particularly, such compositions and methods target miR-29a function in myeloid cell proliferation disorders. Also provided are methods for diagnosing risk or presence of a myeloid cell proliferation disorder in a subject.07-21-2011
20110190383Diagnostic, Prognostic and Therapeutic Uses of MIRs in Adaptive Pathways and/or Disease Pathways - Described herein are methods and compositions for the diagnosis, prognosis and treatment of various adaptive and/or of disease pathways by examining samples containing one or more miRs therein, and by formulating therapeutic agents therefrom.08-04-2011
20110201671Compositions And Methods For Inhibiting Expression Of A Gene From The Ebola Virus - The invention relates to a double-stranded ribonucleic acid (dsRNA) FOR INHIBITING THE expression of a gene from the Ebola virus.08-18-2011
20110201670OLIGORIBONUCLEOTIDES AND METHODS OF USE THEREOF FOR TREATMENT OF FIBROTIC CONDITIONS AND OTHER DISEASES - The invention relates to a double-stranded compound, preferably an oligoribonucleotide (siRNA), which down-regulates the expression of a human TGaseII gene at the post-transcriptional level. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating a patient suffering from a fibrotic disease such as pulmonary, kidney and liver fibrosis or ocular, scarring comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The invention also relates to treatment of fibrotic and other diseases by use of antibodies to TGaseII polypeptide.08-18-2011
20110201668REGULATION OF NEUROTRANSMITTER RELEASE THROUGH ANION CHANNELS - A novel use of anion channels, preferably Ca2+-activated anion channels (CAACs), in regulating release of neurotransmitters from neurons and/or astrocytes is provided. More specifically, CAAC activity regulators, agents for regulating neurotransmitter release comprising such CAAC activity regulators, and methods of screening agents for regulating neurotransmitter release using CAAC as a target.08-18-2011
20110201667COMPOSITIONS AND METHODS FOR SILENCING EBOLA VIRUS GENE EXPRESSION - The present invention provides compositions comprising therapeutic nucleic acids (e.g., interfering RNA such as siRNA) that target Ebola virus (EBOV) gene expression and methods of using such compositions to silence EBOV gene expression. More particularly, the invention provides unmodified and chemically modified interfering RNA which silence EBOV gene expression and methods of use thereof, e.g., for preventing or treating EBOV infections caused by one or more EBOV species such as Zaire EBOV. The invention also provides serum-stable nucleic acid-lipid particles comprising one or more interfering RNA molecules, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. Methods of silencing EBOV gene expression by administering one or more interfering RNA molecules to a mammalian subject are also provided.08-18-2011
20100029747METHODS AND COMPOSITIONS FOR INHIBITING THE FUNCTION OF POLYNUCLEOTIDE SEQUENCES - A therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell includes an agent that provides to a mammalian cell an at least partially double-stranded RNA molecule comprising a polynucleotide sequence of at least about 200 nucleotides in length, said polynucleotide sequence being substantially homologous to a target polynucleotide sequence. This RNA molecule desirably does not produce a functional protein. The agents useful in the composition can be RNA molecules made by enzymatic synthetic methods or chemical synthetic methods in vitro; or made in recombinant cultures of microorganisms and isolated therefrom, or alternatively, can be capable of generating the desired RNA molecule in vivo after delivery to the mammalian cell. In methods of treatment of prophylaxis of virus infections, other pathogenic infections or certain cancers, these compositions are administered in amounts effective to reduce or inhibit the function of the target polynucleotide sequence, which can be of pathogenic origin or produced in response to a tumor or other cancer, among other sources.02-04-2010
20100029746Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes - The invention relates to one or more inhibitors, in particular siRNAs, which down-regulate the expression of human pro-apoptotic genes. The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating or preventing the incidence or severity of hearing impairment (or balance impairment), particularly hearing impairment associated with cell death of the inner ear hair cells or outer ear hair cells, comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient.02-04-2010
20100022623Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.01-28-2010
20100022624Methods of Treating Smooth Muscle Cell Disorders - The present invention provides methods of detecting cells showing smooth muscle differentiation. The present invention further provides methods of detecting tumor cells. The present invention further provides compositions and methods for treating smooth muscle cell disorders.01-28-2010
20100022625Structural-based inhibitors of the glutathione binding site in aldose reductase, methods of screening therefor and methods of use - Provided herein is a crystallized ternary structure of aldose reductase (AR) bound to NADPH and γ-glutamyl-S-(1,2-dicarboxyethyl)cysteinylglycine (DCEG). Also provided are specific inhibitors of glutathione-aldehyde binding to aldose reductase which are designed via at least computer modeling of the ternary AR:NADPH:DCEG structure and methods of designing and of screening the inhibitors for inhibition of glutathione-aldehyde binding to aldose reductase. In addition methods of treating a pathophysiological state or symptoms thereof resulting from aldose reductase-mediated signaling in a cytotoxic pathway using a small interfering RNA (siRNA) or the designed inhibitors.01-28-2010
20100022620EPITOPE REDUCTION THERAPY - The present invention provides the use of an inhibitor of glycolipid biosynthesis in the manufacture of a medicament for the treatment of a glycolipid-mediated autoimmune disease.01-28-2010
20100022622Dbait and its Standalone Uses Thereof - The invention relates to compositions and methods for interfering with the DNA repair of double strand breaks (DSBs). The invention discloses double-stranded nucleic acid molecules that act as baits and hijack the holocomplex of enzymes responsible of DNA DSB sensing, signaling and/or repair pathways, in particular the non-homologous end joining (NHEJ) pathway of DSB repair. The invention discloses the use of these molecules as a standalone anticancer drug in an efficient amount to be introduced in the tumor cell nuclei in order to trigger their death.01-28-2010
20100022619COMPOSITIONS AND THEIR USES DIRECTED TO PTPR ALPHA - Disclosed herein are compounds, compositions and methods for modulating the expression of PTPR.alpha. in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of airway hyperresponsiveness and pulmonary inflammation by administration of antisense compounds targeted to PTPR.alpha.01-28-2010
20110218231Combination of Immuno Gene Therapy and Chemotherapy for Treatment of Cancer and Hyperproliferative Diseases - Pharmaceutical compositions comprising a nucleic acid, a gene delivery polymer, and at least one adjunctive chemotherapeutic drug for the treatment of mammalian cancer or hyperproliferative disorders and methods of using thereof for the treatment of mammalian cancer or hyperproliferative disorders by intratumoral, intraperitoneal or systemic injection.09-08-2011
20110218230NATRIURETIC PEPTIDE RELATED FRAGMENT IN CARDIOVASCULAR DISEASE - This disclosure provides an intracellular fragment of natriuretic peptide receptor A (NPRA), referred to herein as soluble natriuretic peptide receptor-related fragment (sNRF). It is shown herein that sNRF causes NP resistance. Based on these observations, methods of treating a cardiovascular disorder by inhibiting the activity of sNRF are disclosed. Assays are provided that use sNRF to screen agents for their ability to increase the biological activity of an NPR, for example agents that increase the sensitivity of NPR for NPs (such as atrial natriuretic peptide, ANP), or that decrease growth factor deleterious effects, or combinations thereof. Also provided are agents identified using the disclosed assays, and methods of using the agents, for example to treat or diagnose a cardiovascular disorder, such as heart failure.09-08-2011
20110218229SIRT1 Modulation of Adipogenesis and Adipose Function - SIRT1 regulates the physiology of cells of the adipocyte lineage. Modulators of SIRT1 activity can be used to ameliorate, treat, or prevent diseases and disorders associated with adipose physiology, e.g., obesity, an obesity-related disease, or a fat-related metabolic disorder.09-08-2011
20100016405Compositions and Methods for Inhibiting Expression of the MYC Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the MYC gene (MYC gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the MYC gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by MYC gene expression and the expression of the MYC gene using the pharmaceutical composition.01-21-2010
20110021602GRAFT POLYMERS FOR ENHANCED INTRACELLULAR DELIVERY OF ANTISENSE MOLECULES - Innovative graft polymers designed for the efficient delivery of antisense molecules into biological cells and for maintaining the biological activity of these molecules while in serum and other aqueous environments are provided. Such polymers may comprise an anionic graft polymer comprising an anionic polymer backbone with pendant carboxylic acid groups and pendant chains comprising amphipathic or hydrophilic polymers covalently bonded to a portion of said pendant carboxylic acid groups. Antisense molecule delivery vectors comprising such polymers in combination with cationic agents for delivery of antisense molecules are also disclosed.01-27-2011
20100240732APTAMER-TARGETED SIRNA TO PREVENT ATTENUATION OR SUPPRESSION OF A T CELL FUNCTION - Compositions for countering immune attenuating/suppressive pathways comprise targeting agents or aptamer targeted RNAi-mediated gene silencing (siRNA/shRNA). These compositions have broad applicability in the treatment of many diseases.09-23-2010
20100216863Susceptibility Gene for Myocardial Infarction, Stroke, and PAOD; Methods of Treatment - Polymorphisms in the FLAP and LTA4H gene are shown by genetic association analysis to be susceptibility markers for myocardial infarction (MI) and ACS, as well as stroke and PAOD. Pathway targeting for treatment and diagnostic applications in identifying those who are at risk of developing MI, ACS, stroke or PAOD, in particular are described. The invention also provides methods of prophylaxis therapy for MI in human subjects having a race including black African ancestry by administering to the subject a composition comprising a therapeutically effective amount of MI therapeutic agent that inhibits leukotriene synthesis in vivo. The invention also provides for compositions comprising a leukotriene synthesis inhibitor and a statin and methods of using these compositions to reduce C-reactive protein in a human subject at risk of MI, ACS, stroke and/or PAOD.08-26-2010
20100298410MICROMIRs - The present invention relates to very short heavily modified oligonucleotides which target and inhibit microRNAs in vivo, and their use in medicaments and pharmaceutical compositions.11-25-2010
20110077286OLIGONUCLEOTIDE DUPLEXES COMPRISING DNA-LIKE AND RNA-LIKE NUCLEOTIDES AND USES THEREOF - Novel oligonucleotide pairs which can form a duplex comprising one or more DNA-like nucleotides (e.g., 2′-substituted arabinonucleotides (ANA)); in combination with one or more RNA-like nucleotides (e.g., 2′-substituted ribonucleotides (RNA) and/or locked nucleic acid nucleotides (LNA)), are disclosed. The use of such oligonucleotide duplexes, such as for silencing the expression of a nucleic acid or gene of interest using small interfering RNA (siRNA) technologies, is also disclosed.03-31-2011
20110118333Use on Minicircle vectors for cardiac gene therapy - Compositions and methods are provided for the treatment of an ischemic cardiovascular condition by providing a patient with a novel non-viral minicircle DNA vector comprising polynucleotide sequences that potentiate HIF-1 activity, including RNAi or antisense agents selective for proteins involved in HIF1 inactivation.05-19-2011
20110118337Method of Using Compositions Comprising MIR-192 and/or MIR-215 for the Treatment of Cancer - The invention provides methods and compositions for inhibiting the proliferation of mammalian cells. In some embodiments, the methods comprise contacting mammalian cells with an effective amount of at least one small interfering nucleic acid (siNA) agent that inhibits the level of expression of at least two miR 192 family responsive genes selected from the group consisting of SEPT 10, LMNB2, HRH1, HOXA10, ERCC3, MIS12, MPHOSPHI1, CDC7, SMARCB1, MAD2L1, DTL, RAC-GAP1, MCM10, PIM1, DLG5, BCL2, CUL5, and PRPF38A.05-19-2011
20110118334ANTISENSE ANTIVIRAL COMPOUND AND METHOD FOR TREATING INFLUENZA VIRAL INFECTION - The present invention relates to antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Orthomyxoviridae family and in the treatment of a viral infection. The compounds are particularly useful in the treatment of influenza virus infection in a mammal. Exemplary antisense antiviral compounds are substantially uncharged, or partially positively charged, morpholino oligonucleotides having 1) a nuclease resistant backbone, 2) 12-40 nucleotide bases, and 3) a targeting sequence of at least 12 bases in length that hybridizes to a target region selected from the following: a) the 5′ or 3′ terminal 25 bases of the negative sense viral RNA segment of Influenzavirus A, Influenzavirus B and Influenzavirus C; b) the terminal 30 bases of the 5′ or 3′ terminus of the positive sense vcRNA; c) the 45 bases surrounding the AUG start codon of an influenza viral mRNA and; d) 50 bases surrounding the splice donor or acceptor sites of influenza mRNAs subject to alternative splicing.05-19-2011
20110118336Microrna-21 antagonists and its target PDCD4 for use in the treatment of a glioma - The present invention embraces microRNA-21 antagonists and activators of Programmed Cell Death 4 for use in decreasing glial tumor cell proliferation and treating glioma.05-19-2011
20090298915TOPICAL DRUG DELIVERY - Poly-pseudo-lysine conjugates have been shown to be able to penetrate into human skin and are proposed for both therapeutic and cosmetic treatments by topical application, e.g. change of skin pigmentation.12-03-2009
20110306654Methods for identifying analgesic agents - The present invention relates to the discovery that mutations in SCN9A are causative of Congenital Indifference to Pain (CIP) in humans. The invention also relates to methods of utilizing the SCN9A gene and expression products thereof for the screening and identification of therapeutic agents, including small organic compounds, which are selective for SCN9A, and are useful in the treatment of pain and other disorders. The invention also relates to methods of using these compounds to treat or otherwise ameliorate such disorders.12-15-2011
20100010064Cancer treatment by combined inhibition of proteasome and telomerase activities - A method and kit for inhibiting the proliferation of cancer cells are disclosed, based on a combination of a proteasome inhibitor and a telomerase inhibitor. When used in cancer therapy, the two compounds in combination enhance the anti-cancer treatment efficacy obtained with the proteasome inhibitor alone or the telomerase inhibitor alone. Preferably, efficacy is supraadditive or synergistic in nature relative to the combined effects of the individual agents, with minimal exacerbation of side effects.01-14-2010
20100305188NUCLEIC ACID CAPABLE OF REGULATING THE PROLIFERATION OF CELL - Provided are an agent for suppressing or promoting cell proliferation, a diagnostic reagent or therapeutic drug for a disease resulting from a cell proliferation abnormality, an agent for inducing apoptosis, an agent for suppressing or promoting the expression of a target gene for a nucleic acid such as a microRNA, and a method of suppressing or promoting cell proliferation.12-02-2010
20100298403MODIFIED POLY(PROPYLENE-IMINE) DENDRIMERS AND THEIR USE AS TRANSFECTION AGENTS FOR AMIONIC BIOACTIVE FACTORS ( as amended - The present invention is concerned with modified poly-(propylene imine) dendrimers, comprising cationic internal ammonium groups and external non-toxic endgroups, pharmaceutical compositions comprising said dendrimers, methods for the production of said dendrimers and their use as transfections agents for anionic bioactive therapeutic factors, for use in gene therapy, in particular for the treatment of cancer. The modified poly-(propylene imine) dendrimer of generation 1, 2, 3, 4 or 5, also comprising incomplete dendrimers and mixtures thereof, comprising external end groups and internal amine functions are characterized in that: 11-25-2010
20090318534METHODS AND COMPOSITIONS FOR THE TREATMENT OF SKIN DISEASES AND DISORDERS - The disclosure demonstrates the role of cathelicidin, serine protease and/or vitamin D3 in rosacea pathology.12-24-2009
20090247611METHOD OF TREATING A CANCER WITH A SURVIVIN ANTISENSE OLIGONUCLEOTIDE AND PACLITAXEL - Provided is a method of treating cancer of the stomach, comprising administering to a patient a therapeutically effective combination of a Survivin antisense oligonucleotide and paclitaxel.10-01-2009
201001908375'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom - The present invention provides 5′-substituted-2′-F nucleoside analogs and oligomeric compounds comprising these nucleoside analogs. In one preferred embodiment the nucleoside analogs have either (R) or (5)-chirality at the 5′-position. These nucleoside analogs are expected to be useful for enhancing properties of oligomeric compounds including nuclease resistance.07-29-2010
20130197061AGENT FOR SUPPRESSING EXPRESSION OF DOMINANT MUTANT GENE - An RNAi molecule that can selectively and effectively suppress only the expression of a particular dominant mutant gene, while permitting the expression of the wild-type gene or a desired mutant gene, and a design method thereof is presented.08-01-2013
20110039912Therapeutic Agent for Neuroblastoma Targeting ARID3b - It is intended to provide a therapeutic agent for neuroblastoma. More particularly, it is intended to provide the therapeutic agent for neuroblastoma containing an ARID3b inhibitor.02-17-2011
20110152347Methods and compositions for controlling efficacy of RNA silencing - Based at least in part on an understanding of the mechanisms by which small RNAs (e.g., naturally-occurring miRNAs) mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, the effect of the position of the mismatches, etc. Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing.06-23-2011
20110152344Inhibition of filovirus entry into cells and uses thereof - The present invention discloses method to treat infections caused by filovirus. Such a method comprises blocking the PI3 kinase pathway or the calcium-associated pathway at the gene or protein level. Also disclosed herein are the compounds useful in the treatment of filoviral infection.06-23-2011
20100016406Antisense RNA for Treating Cancer and Inhibition of Metastasis and Vectors for Antisense Sequestration - Provided is the use of antisense RNA and methods for the treatment, diagnosis and prophylaxis of cancer comprising administering said antisense RNA, particularly miRs 15 and 16 to a patient in need thereof.01-21-2010
20110306651RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a 12-15-2011
20110306655METHODS FOR IDENTIFYING AND COMPOUNDS USEFUL FOR THE DIAGNOSIS AND TREATMENT OF DISEASES INVOLVING INFLAMMATION - The present invention relates to agents, and methods for identifying compounds, which agents and compounds result in the inhibition of the maturation of dendritic cells. In addition, the invention relates to compositions and methods for the use thereof in treating conditions that are characterized by maturation of dendritic cells including infections, allograft reactions, inflammation, allergic and autoimmune diseases, and cancer.12-15-2011
20110306653STABILIZATION METHOD OF FUNCTIONAL NUCLEIC ACID - This invention is intended to enhance and improve the resistance of a single- or double-stranded nucleic acid fragment comprising a base sequence of a functional nucleic acid to degradation by nucleolytic enzymes in a simple and cost-effective manner. The single- or double-stranded nucleic acid fragment comprises, ligated to at least one end thereof, hairpin-shaped DNA comprising: (A) a nucleic acid region comprising 2 to 5 arbitrary nucleotides; (B) a nucleic acid region comprising a “gna” or “gnna” base sequence, wherein each “n” represents “g”, “t”, “a”, or “c”, a base analogue, or a modified base; and (C) a nucleic acid region comprising a base sequence complementary to the nucleic acid region (A), sequentially from the 5′ end toward the 3′ end.12-15-2011
20110306652COMPOSITIONS AND THEIR USES DIRECTED TO HUNTINGTIN - Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.12-15-2011
20120041048COMPOSITIONS AND METHODS RELATING TO miR-31 - MicroRNA-31 (miR-31), targets of miR-31, the role of miR-31 in inhibiting tumor metastasis, and the role of miR-31 target genes in promoting tumor metastasis are disclosed.02-16-2012
20120041049DESIGN OF SMALL-INTERFERING RNA - The present invention relates to small-interfering RNA molecules displaying an increased thermodynamic stability at the 3′ end of the antisense strand (guide strand) and the 5′ end of the sense strand (passenger strand), respectively, in comparison to the base pairing in the seed region. The siRNAs of the present invention display an increased knock-down activity against targeted genes and show an improved resistance to RNAses, in particular serum RNAses. The present invention also relates to a method for the production of the siRNA molecules, a method of target-specific RNA interference making use of the improved siRNA molecules of the invention and pharmaceutical compositions containing the siRNA molecules.02-16-2012
20100331392ANTIDOTES TO ANTISENSE COMPOUNDS - The present invention relates to antisense antidote compounds and uses thereof. Such antidote compounds reduce the magnitude and/or duration of the antisense activity of an antisense compound.12-30-2010
20110060032LIPID ENCAPSULATING INTERFERING RNA - The present invention provides lipid-based formulations for delivering, e.g., introducing, nucleic acid-lipid particles comprising an interference RNA molecule to a cell, and assays for optimizing the delivery efficiency of such lipid-based formulations.03-10-2011
20120077862ANTISENSE MODULATION OF PTP1B EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of PTP1B. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding PTP1B. Methods of using these compounds for modulation of PTP1B expression and for treatment of diseases associated with expression of PTP1B are provided.03-29-2012
20090215861Antisense oligonucleotides for treating allergy and neoplastic cell proliferation - Antisense oligonucleotides for treating and/or preventing at least one of asthma, allergy, hypereosinophilia, general inflammation and cancer are provided. The oligonucleotides are directed against nucleic acid sequences coding for a receptor selected from the group consisting of a CCR3 receptor and a common sub-unit of IL-3, IL-5 and GM-CSF receptors.08-27-2009
20090247604RNAi Therapeutics for Treatment of Eye Neovascularization Diseases - Compositions and methods for treating ocular disease are provided. Specifically, siRNA molecules and mixtures of siRNA molecules are provided that inhibit angiogenesis and/or neovascularization. The compositions and methods are suitable for treating ocular diseases associated with angiogenesis and/or neovascularization.10-01-2009
20110015252LIPID FORMULATED DSRNA TARGETING THE PCSK9 GENE - This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene.01-20-2011
20100168210T-Cell Cytokine-Inducing Surface Molecules and Methods of Use - The invention provides cytokine modulators and methods for using the same to modulate cytokine production in monocyte lineage-derived cells. In particular, cytokine modulators of the invention selectively bind to a T-cell cytokine-inducing surface molecule (TCISM)-ligand of T lymphocytes or the corresponding TCISM-receptor of monocyte lineage-derived cells, thereby modulating cytokine production in monocyte lineage-derived cells.07-01-2010
20090318535BETA -TrCP1, BETA -TrCP2 AND RSK1 OR RSK2 INHIBITORS AND METHODS FOR SENSITIZING TARGET CELLS TO APOPTOSIS - The invention relates to modulating BimEL levels (Bcl-2-Interacting Mediator of cell death, Extra Long isoform) to sensitize cancer cells to cell death or apoptosis. In certain embodiments, the invention relates to increasing BimEL levels. In certain embodiments, the invention relates to inhibitors of at least one of β-TrCP1/2 or RSK1/2 proteins that sensitize tumor cells to chemotherapy-induced death or apoptosis. Additionally, the invention relates to cancer therapies, diagnostics, and methods for identifying novel drugs or drug candidates for increasing BimEL levels.12-24-2009
20110060033FERTILITY RESTORER GENE AND FERTILITY RESTORATION METHOD FOR CW-TYPE MALE STERILE CYTOPLASM OF RICE - Mainly provided is a technique for directly identifying the genotype at locus Rf17 based on the specific base sequence data thereof. Also provided is a technique for artificially constructing a fertility-restored line. A method of restoring the fertility of CW-type cytoplasmic male sterile rice by inhibiting or reducing the expression of a gene comprising the base sequence represented by SEQ ID NO:2 in the above-described rice, and a method for determining the presence or absence of gene Rf17, which is a fertility restorer gene for CW-type cytoplasmic male sterility, comprising identifying a single nucleotide polymorphism (SNP) in the base at the 1812 position of the base sequence represented by SEQ ID NO:1 in the rice to be examined.03-10-2011
20100137411RAS-MEDIATED EPIGENETIC SILENCING EFFECTORS AND USES THEREOF - The invention relates to methods for inhibiting gene silencing, methods for inhibiting cell proliferation, methods for inhibiting Ras mediated tumor growth, methods for screening for regulators of FAS expression, and methods for identifying inhibitors of Ras mediated tumor growth.06-03-2010
20120041053DELIVERY OF dsRNA TO ARTHROPODS - The invention is to methods of gene silencing in arthropods using dsRNA. The method is include contacting the arthropod with, and/or directly feeding the arthropod, the dsRNA to the arthropods to deliver the dsRNA to arthropod tissues. It is envisaged that the methods of the invention will have use in determining the biological function of genes in arthropods. Methods of pest control of arthropods, and of protecting arthropods against parasites and predators are provided. Transgenic arthropods expressing dsRNA molecules are also provided by the present invention.02-16-2012
20110028532Methods of treating eye diseases in diabetic patients - The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of the RTP801 gene and/or protein.02-03-2011
20120041054COMPOSITIONS AND METHODS FOR THE TREATMENT OF SEPSIS AND OTHER DISORDERS INVOLVING PHOSPHOLIPASE A2 INDUCTION - The present invention provides antisense oligomers to PLA02-16-2012
20120041052Compositions and Methods to Treat Muscular & Cardiovascular Disorders - The present invention relates to a novel microRNA, mir-208-2, implicated in muscular and cardiovascular disorders. The present invention also relates to oligonucleotide therapeutic agents (antisense oligonucleotides and/or double stranded oligonucleotides such as dsRNA) and their use in the treatment of muscular and cardiovascular disorders resulting from dysregulation of mir-208-2.02-16-2012
20120041051Compositions And Methods For Inhibiting Expression Of MIG-12 Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting MIDI interacting G12-like protein (MIG 12) gene, and methods of using the dsRNA to inhibit expression of MIG 12.02-16-2012
20120041047Injectable Pharmaceutical Composition for Preventing, Stabilizing and/or Inhibiting Pathological Neovascularization-Related Conditions - This invention relates to a pharmaceutical composition for the treatment and/or prevention of at least one pathological neovascularization-related conditions of the interior of the eye, the composition comprising a therapeutically effective amount of an antisense oligonucleotide having the sequence SEQ ID NO: 1:02-16-2012
20110105590Compositions And Methods For Inhibiting Expression Of Anti-Apoptotic Genes - The present invention relates to an isolated double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of bcl-2, where the antisense strand comprises a sequence that comprises a region of complementarity which is substantially complementary to at least a part of an mRNA encoding bcl-2. The dsRNA, upon contact with a cell expressing the bcl-2, inhibits expression of the bcl-2 gene by at least 20%. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier. The invention further relates to a vector for inhibiting expression of bcl-2 in a cell, where the vector comprises a regulatory sequence operably linked to a nucleotide sequence that encodes at least one strand of the dsRNA.05-05-2011
20100317719Antisense Oligonucleotides (ODN) Against SMAD7 and Uses Thereof in Medical Field - The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.12-16-2010
20110086902COMPOUNDS FOR THE MODULATION OF BETA-CATENIN EXPRESSION - The invention relates to oligomer compounds (oligomers), which target beta-catenin mRNA in a cell, leading to reduced expression of beta-catenin. Reduction of beta-catenin expression is beneficial for a range of medical disorders, such as hyperproliferative disorders, such as cancer. The invention provides therapeutic compositions comprising oligomers and methods for modulating the expression of beta-catenin using said oligomers, including methods of treatment.04-14-2011
20110313025METHODS AND COMPOSITIONS INVOLVING MIRNA AND MIRNA INHIBITOR MOLECULES - The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.12-22-2011
20110313024RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression and/or activity. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.12-22-2011
20100048671INHIBITION OF THE 44 KILODALTON ISOFORM OF PIM-1 KINASE RESTORES APOPTOSIS INDUCED BY CHEMOTHERAPEUTIC DRUGS IN CANCER CELLS - The present invention relates to a newly discovered 44 kD isoform of Pim-1 kinase made in human cells, and to the gene and messenger RNA for the 44 kilodalton isoform. The invention further describes methods and compounds for treating, especially prostate and hematopoietic cancer, by inhibiting expression of the 44 kD isoform of Pim-1 kinase, or its ability to phosphorylate Etk kinase and breast cancer resistance protein (BCRP).02-25-2010
20120041050ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.02-16-2012
20090275634ANTISENSE OLIGONUCLEOTIDES AGAINST ACETYLCHOLINESTERASE FOR TREATING INFLAMMATORY DISEASES - The present invention relates to novel uses of antisense oligonucleotides targeted to the coding region of acetylcholinesterase (AChE) for treating inflammatory disorders other than inflammatory disorders of the central nervous system or the peripheral nervous system innervating voluntary muscles. More particularly, the present invention relates to uses of antisense oligodexoynucleotides targeted to AChE mRNA for treating inflammatory disease of the gastrointestinal tract including inflammatory bowel disease.11-05-2009
20100280100TREATMENT OF HEMOGLOBIN (HBF/HBG) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO HBF/HBG - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Hemoglobin (HBF/HBG), in particular, by targeting natural antisense polynucleotides of Hemoglobin (HBF/HBG). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of HBF/HBG.11-04-2010
20100168203ADENYLYL CYCLASES AS NOVEL TARGETS FOR ANTIBACTRIAL INTERVENTIONS - The present invention relates to a method of preventing or treating a disease caused by bacterial infection by administering an effective amount of a modulator of bacterial adenylyl cyclase. The invention also provides pharmaceutical compositions useful for preventing or treating a disease, with the compositions containing a therapeutically effective amount of a modulator of bacterial adenylyl cyclase. The invention also provides screening methods for identifying selective modulators of bacterial adenylyl cyclase that do not substantially modulate adenylyl cyclase of the subject. The invention also provides methods for culturing bacterial pathogens and methods for inducing the pathogenic state in vitro.07-01-2010
20110071210METHODS AND COMPOSITIONS FOR THE TREATMENT OF CANCER OR OTHER DISEASES - The present invention relates to methods and compositions for the treatment of diseases, including cancer, infectious diseases and autoimmune diseases. The present invention also relates to methods and compositions for improving immune function. More particularly, the present invention relates to multifunctional molecules that are capable of being delivered to cells of interest for the treatment of diseases and for the improvement in immune function.03-24-2011
20120208862RIP140 REGULATION OF GLUCOSE TRANSPORT - Inhibition of RIP140 increases glucose transport. Compounds that inhibit RIP140 expression or activity are useful for treating disorders associated with aberrant glucose transport (e.g., diabetes), treating obesity, increasing metabolism (e.g., fatty acid metabolism), and increasing brown fat.08-16-2012
20120046339Novel chorismate mutase gene from the potato cyst nematode globodera rostochiensis - The nucleotide sequence of a 992 bp region of cDNA and the nucleotide sequence of a 1973 bp (or a 1913 bp) of genomic DNA of the Gr-cm-1 gene were determined for 02-23-2012
20120046342OLIGONUCLEOTIDE COMPRISING AN INOSINE FOR TREATING DMD - The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.02-23-2012
20120046340Down Regulation of the Gene Expression by Means of Nucleic Acid-Loaded Virus-Like Particles - The present invention relates to compositions of virus-like particles for the introduction of RNA-interference (RNAi-) inducing molecules into eukaryotic cells and methods for the cell type-specific transduction of a plurality of eukaryotic cells with RNAi-inducing molecules. The present invention furthermore relates to methods for a diagnosis, prevention and/or treatment of diseases or disease states associated with an increased expression rate of at least one endogenous gene, and/or with the undesired expression of at least one endogenous gene and/or foreign nucleic acids, in particular viral nucleic acids.02-23-2012
20120065246RNA MOLECULES AND THERAPEUTIC USES THEREOF - The invention relates to double-stranded RNA molecules in which each strand of said molecule possesses: (a) sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof; and (b) sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex; and in which at least one strand of said molecule possesses: (c) a seed region of complementarity to at least one seed site present in a 3′ untranslated region of at least one target mRNA molecule. The invention also relates to an algorithm for the design of a double-stranded RNA molecule in which each strand of said molecule possesses: (a) sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof; and (b) sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex; and in which at least one strand of said molecule possesses: (c) a seed region of complementarity to at least one seed site present in a 3′ untranslated region of at least one target mRNA molecule; wherein said algorithm comprises the steps: (i) input a population of mRNA sequences transcribed from one or more genes of interest; (ii) identify all subsequences of at least 12 nucleotides in length within the population of step (i) which are complementary to another subsequence of at least 12 nucleotides in length in the population; (iii) determine a list of candidate bi-functional double-stranded RNA molecules, said list comprising the double-strand RNA duplexes comprising the two complementary subsequences of step (ii); and (iv) sort the list of candidate double-stranded RNA molecules of step (iii) based on their potential to cause translational suppression.03-15-2012
20120046341FUS/TLS-BASED COMPOUNDS AND METHODS FOR DIAGNOSIS, TREATMENT AND PREVENTION OF AMYOTROPHIC LATERAL SCLEROSIS AND RELATED MOTOR NEURON DISEASES - The invention provides novel FUS/TLS nucleic acids and proteins that comprise one or more genetic markers (for example, single nucleotide polymorphisms) and methods of use thereof including methods relating to the diagnosis of ALS or other related motor neuron disease by virtue of the presence of the mutant FUS/TLS sequence(s).02-23-2012
20120046343MULTI-TARGETING SHORT INTERFERING RNAs - The present invention relates to novel short interfering RNA (siRNA) molecules that are multi-targeted. More specifically, the present invention relates to siRNA molecules that target two or more sequences. In one embodiment, multi-targeting siRNA molecules are designed to incorporate features of siRNA molecules and features of micro-RNA (miRNA) molecules. In another embodiment, multi-targeting siRNA molecules are designed so that each strand is directed to separate targets.02-23-2012
20120004278LINC RNAS IN CANCER DIAGNOSIS AND TREATMENT - Long non-coding RNAs (lincRNAs), a relatively recently recognized class of widely transcribed genes, are thought to affect chromatin state and epigenetic regulation, but their mechanisms of action and potential roles in human disease are poorly understood. The present invention shows that long non-coding RNAs in the human HOX loci are systematically dysregulated during breast cancer progression, and that expression levels of the lincRNA termed HOTAIR can predict cancer metastasis. Elevated levels of HOTAIR can lead to altered patterns of Polycomb binding to the genome. These findings indicate that lincRNAs have active roles in modulating the cancer epigenome and may be important targets for cancer diagnosis and therapy.01-05-2012
20120004281RNA Interference Mediated Inhibition of the Nerve Growth Factor Beta Chain (NGFB) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of NGFβ gene expression and/or activity, and/or modulate a NGFβ gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against NGFβ gene expression.01-05-2012
20120004280RNA Interference Mediated Inhibition of the High Affinity 1 gE Receptor Alpha Chain (FC Epsilon R1 Alpha) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of FCεR1α gene expression and/or or activity, and/or modulate a FCεR1α gene expression pathway. Specifically, the invention relates to doublestranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against FCεR1α gene expression.01-05-2012
20120004276RNA ANTAGONIST COMPOUNDS FOR THE INHIBITION OF EXPRESSION OF MITOCHONDRIAL GLYCEROL-3 PHOSPHATE ACYLTRANSFERASE 1 (MTGPAT1) - The present invention relates to oligomer compounds (oligomers), which target mtGPAT1mRNA in a cell, leading to reduced expression of mtGPAT1. Reduction of mtGPAT1 expression is beneficial for the treatment of certain medical disorders, such as overweight, obesity, fatty liver, hepatosteatosis, non alcoholic fatty liver disease (NAFLD), non alcoholic steatohepatitis (NASH), insulin resistance, and non insulin dependent diabetes mellitus (NIDDM).01-05-2012
20120004282RNA Interference Mediated Inhibition of the Intercellular Adhesion Molecule 1 (ICAM-1) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of ICAM-1 gene expression and/or activity, and/or modulate a ICAM-1 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against ICAM-1 gene expression.01-05-2012
20120208864COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF GCGR - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.08-16-2012
20120208861IFN TYPE-I PRODUCTION INHIBITOR AND METHOD FOR SCREENING FOR SAME - It has been found that Spi-B, in cooperation with IRF-7, induces type I IFN production. This invention is based on the finding, and provides a type I IFN production inhibitor comprising an antisense nucleic acid or siRNA against Spi-B, or an expression vector capable of expressing the same; a screening method for a substance capable of inhibiting type I IFN production, comprising selecting a substance that suppresses the expression or function of Spi-B as a substance capable of inhibiting type I IFN production; and a type I IFN production inducer comprising an expression vector capable of expressing Spi-B and an expression vector capable of expressing IRF-7 in combination, and the like.08-16-2012
20120108653OLIGOMERS - Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.05-03-2012
20120108651GENETIC POLYMORPHISMS ASSOCIATED WITH VENOUS THROMBOSIS AND STATIN RESPONSE, METHODS OF DETECTION AND USES THEREOF - The present invention provides compositions and methods based on genetic polymorphisms that are associated with response to statin treatment (particularly for reducing the risk of venous thrombosis). For example, the present invention relates to nucleic acid molecules containing the polymorphisms, variant proteins encoded by these nucleic acid molecules, reagents for detecting the polymorphic nucleic acid molecules and variant proteins, and methods of using the nucleic acid molecules and proteins as well as methods of using reagents for their detection.05-03-2012
20120108648MITOCHONDRIAL FUNCTION-IMPROVING AGENT - It is an object of the present invention to provide a novel mitochondrial function-improving agent and a novel PGC-1α expression inducing agent. The present invention provides a mitochondrial function-improving agent and a PGC-1α expression inducing agent each of which comprises a lysine-specific demethylase-1 (LSD-1) inhibitor.05-03-2012
20110054003ANTISENSE OLIGONUCLEOTIDE MODULATION OF STAT3 EXPRESSION - Compounds, compositions and methods are provided for inhibiting the expression of human STAT3. The compositions comprise antisense oligonucleotides targeted to nucleic acids encoding STAT3. Methods of using these oligonucleotides for inhibition of STAT3 expression and for promotion of apoptosis are provided. Methods for treatment of diseases, particularly inflammatory diseases and cancers, associated with overexpression or constitutive activation of STAT3 or insufficient apoptosis are also provided.03-03-2011
20120172412In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages - The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.07-05-2012
20120108647THERAPEUTIC USES OF INHIBITORS OF RTP801L - The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases respiratory conditions and hearing disorders based upon inhibition of the RTP801L gene and/or protein.05-03-2012
20120108646RNAi Modulation Of TGF-BETA And Therapeutic Uses Thereof - The present invention concerns methods of treatment using transforming growth factor beta (TGF-beta) modulators. More specifically, the invention concerns methods of treating disorders associated with undesirable TGF-beta signaling, by administering short interfering RNA which down-regulate the expression of TGF-beta, and agents useful therein.05-03-2012
20120010272RNA Interference Mediated Inhibition of Apoptosis Signal-Regulating Kinase 1 (ASK1) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of ASK1 gene expression and/or activity, and/or modulate a ASK1 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against ASK1 gene expression.01-12-2012
20120172409METHODS AND KITS FOR LINKING POLYMORPHIC SEQUENCES TO EXPANDED REPEAT MUTATIONS - Methods and kits are provided for determining which single nucleotide polymorphism (“SNP”) variant of an allele of a heterozygous patient is on the same allele as a disease-causing mutation. Also, provided are kits for multi-SNP diagnosis and treatment, targeting combinations of SNPs having greater joint prevalence of heterozygosity in Huntington's population than individual SNPs considered individually.07-05-2012
20120010267RNAi MODULATION OF MLL-AF4 AND USES THEREOF - The invention relates to compositions and methods for modulating the expression of the MLL-AF4 fusion gene, and more particularly to the downregulation of MLL-AF4 by chemically modified oligonucleotides.01-12-2012
20120010271SINGLE-STRANDED NUCLEIC ACID MOLECULE FOR CONTROLLING GENE EXPRESSION - Provided is a novel nucleic acid molecule that can inhibit the expression of a gene and can be produces easily and efficiently. The nucleic acid molecule is a single-stranded nucleic acid molecule including an expression inhibitory sequence that inhibits expression of a target gene. The single-stranded nucleic acid molecule includes, in sequence from the 5′ side to the 3′ side: a 5′ side region (Xc); an inner region (Z); and a 3′ side region (Yc). The inner region (Z) is composed of an inner 5′ side region (X) and an inner 3′ side region (Y) that are linked to each other. The 5′ side region (Xc) is complementary to the inner 5′ side region (X). The 3′ side region (Yc) is complementary to the inner 3′ side region (Y). At least one of the inner region (Z), the 5′ side region (Xc), and the 3′ side region (Yc) includes the expression inhibitory sequence. According to this single-stranded nucleic acid molecule, it is possible to inhibit the expression of the target gene.01-12-2012
20120010270ISOLATED GENOMIC POLYNUCLEOTIDE FRAGMENTS FROM THE p15 REGION OF CHROMOSOME 11 ENCODING HUMAN TUMOR SUPPRESSING SUBTRANSFERABLE CANDIDATE 6 (TSSC6) - Provided herein are isolated genomic polynucleotide fragments from the from the p15 region of chromosome 11 encoding human tumor suppressing subtransferable candidate 6 and methods of use.01-12-2012
20120010269ISOLATED GENOMIC POLYNUCLEOTIDE FRAGMENTS FROM THE p15 REGION OF CHROMOSOME 11 ENCODING HUMAN SMS3 - Provided herein are isolated genomic polynucleotide fragments from the from the p15 region of chromosome 11 encoding human SMS3 (SMS3) and methods of use.01-12-2012
20120010268ISOLATED GENOMIC POLYNUCLEOTIDE FRAGMENTS FROM THE p15 REGION OF CHROMOSOME 11 ENCODING HUMAN ACHAETE-SCUTE HOMOLOG 2 (HASH2) - Provided herein are isolated genomic polynucleotide fragments from the p15 arm of chromosome 11 encoding HASH2 and methods of use.01-12-2012
20120010266TBC1D7 AS TUMOR MARKER AND THERAPEUTIC TARGET FOR CANCER - The present invention relates to the roles played by the TBC1D7 genes in cancer, in particular, lung cancer or esophageal cancer, or carcinogenesis and features a method for treating and/or preventing cancer, in particular, lung cancer or esophageal cancer by administering a double-stranded molecule against one or more of the TBC1D7 genes or a composition, vector or cell containing such a double stranded molecule. The present invention also features methods for diagnosing lung or assessing/determining the prognosis of a patient with lung, especially NSCLC or SCLC, or esophageal cancer, using one or more over-expressed genes selected from among TBC1D7. To that end, TBC1D7 may serve as a novel biomarker for lung cancer or esophageal cancer. Also, disclosed are methods of identifying compounds for treating and preventing lung or esophageal cancer, using as an index for their effect on the over-expression of one or more of TBC1D7 in the lung cancer or esophageal cancer.01-12-2012
20120046345TREATMENT OF DYSTROPHIN FAMILY RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO DMD FAMILY - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Dystrophin family, in particular, by targeting natural antisense polynucleotides of Dystrophin family. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of DMD family.02-23-2012
20100184823dsRNA For Treating Viral Infection - The invention relates to double-stranded ribonucleic acids (dsRNAs) targeting gene expression of phosphatidylinositol 4-kinase (PI4K), in particular human phosphatidylinositol 4-kinase, catalytic, beta polypeptide (PIK4CB) or human phosphatidylinositol 4-kinase, catalytic, alpha polypeptide (PIK4CA), and their use for treating infection by positive stranded RNA viruses such as hepatitis C virus (HCV). Each dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PIK4CB or PIK4CA target mRNA. A plurality of such dsRNA may be employed to provide therapeutic benefit. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, and including a delivery modality such as fully encapsulated liposomes or lipid complexes. The invention further includes methods for treating diseases caused by positive stranded RNA virus infection using the pharmaceutical compositions; and methods for inhibiting the propagation of positive stranded RNA viruses in and between cells.07-22-2010
20090239931RNA INTERFERENCE MEDIATED INHIBITION OF PROTEIN TYROSINE PHOSPHATASE-1B (PTP-1B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating protein tyrosine phosphatase-1B (PTP-1B) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of PTP-1B gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of PTP-1B genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing obesity, insulin resistance, diabetes (eg. type II and type I diabetes) in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of PTP-1B in a cell or tissue, alone or in combination with other treatments or therapies.09-24-2009
20110166205SUBSTITUTED ALPHA-L-BICYCLIC NUCLEOSIDES - The present disclosure describes substituted α-L-bicyclic nucleoside analogs, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, substituted α-L-bicyclic nucleoside analogs are provided, having one or more chiral substituents, that are useful for enhancing properties of oligomeric compounds including binding affinity. In some embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.07-07-2011
20110166204OLIGONUCLEOTIDES TARGETING HUMAN ENDOGENOUS RETROVIRUS 9 (ERV-9) LONG TERMINAL REPEAT (LTR) AND METHODS OF USE - Described herein are oligonucleotides that target the human endogenous retrovirus-9 (ERV-9) long terminal repeat (LTR). The ERV-9 LTR oligonucleotides specifically hybridize with either the coding strand or non-coding strand of ERV-9 LTR. It is disclosed herein that ERV-9 LTR oligonucleotides inhibit the proliferation of cancer cells, including breast cancer, liver cancer, prostate cancer, fibrosarcoma and myeloid cancer cells. Also described herein are methods of treating a subject diagnosed with cancer comprising administering to the subject an ERV-9 LTR oligonucleotide. In some examples, the methods further comprise administering a second therapeutic agent, such as an antisense compound or a chemotherapeutic agent.07-07-2011
20110166199RNAi COMPOUND TARGETED TO THROMBOSPONDIN-1 AND APPLICATIONS THEREOF - The present invention relates to an RNAi compound and an expression plasmid for inhibiting expression of Thrombospondin-1, which comprises a target sequence selected from Thrombospondin-1 gene. The present invention also related to a pharmaceutical composition comprising the RNAi compound and applications thereof. The RNAi compound can reduce the expression of Thrombospondin-1 to activate immune responses. In addition, the present invention also disclosed that an RNAi compound targeted to Thrombospondin-1 gene can delay tumor progression.07-07-2011
20110166200METHODS OF USING MIR210 AS A BIOMARKER FOR HYPOXIA AND AS A THERAPEUTIC AGENT FOR TREATING CANCER - The present invention provides compositions and methods for predicting the hypoxia response in tumor cells, methods for predicting the likelihood of cancer metastasis, and methods for inhibiting tumor cell proliferation using a microRNA comprising miR-210.07-07-2011
20110166198RNA COMPOSITIONS FOR MODULATING IMMUNE RESPONSE - This invention features modified iRNA agents that modulate an immune response, methods of making and identifying iRNA agents that modulate an immune response, and methods of using the iRNA agents to modulate an immune response.07-07-2011
20110166197Antisense Modulation Of Amyloid Beta Protein Expression - Antisense nucleic acids, compositions and methods are provided for modulating the expression of an amyloid beta protein (AβP) portion of the amyloid precursor protein (APP) coding sequence. The compositions comprise antisense nucleic acids targeted to nucleic acids encoding amyloid precursor protein. Methods of using these nucleic acids for modulation of amyloid precursor protein expression and for treatment of diseases and conditions associated with expression of the amyloid beta protein portion of the amyloid precursor protein are provided.07-07-2011
20110166196TREATMENT AND PROPHYLAXIS OF EPILEPSY AND FEBRILE SEIZURES - Provided are methods for treatment and prophylaxis of convulsive disorders and seizures, such as epilepsy and febrile seizures, by modulating TRPV07-07-2011
20110166202FORMOTEROL/STEROID BRONCHODILATING COMPOSITIONS AND METHODS OF USE THEREOF - Bronchodilating compositions and methods are provided. The compositions are intended for administration as a nebulized aerosol. In certain embodiments, the compositions contain formoterol, or a derivative thereof, and a steroidal anti-inflammatory agent. Methods for treatment, prevention, or amelioration of one or more symptoms of bronchoconstrictive disorders using the compositions provided herein are also provided.07-07-2011
20120059043THERAPEUTIC AND DIAGNOSTIC STRATEGIES - The present invention encompasses the finding that microRNAs (miRNAs) regulate certain key proteins involved in DNA repair. In some embodiments, a miRNA suppresses levels and/or activity of one or more DNA repair proteins. In some such embodiments, such suppression renders cells hypersensitive to certain DNA damage agents (e.g., γ-irradiation and genotoxic drugs, among others). The present invention provides various reagents and methods associated with these findings including, among other things, strategies for treating cell proliferative disorders, certain diagnostic systems, etc.03-08-2012
20120016003POT1 ALTERNATIVE SPLICING VARIANTS - The present invention provides methods and compositions for diagnosis and treatment of carcinomas with aberrant expression patterns of POT 1. The invention also provides methods of identifying compounds that may modulate the cellular expression of POT 1. The invention further provides methods for treating subjects suffering from or at risk of developing a colorectal carcinoma.01-19-2012
20120016005PHAGOCYTIC CELL DELIVERY OF RNAI - The present invention provides a particulate delivery system for delivering an RNAi construct to phagocytic cells such as macrophages, comprising various configurations of a complex comprising a phagocytic cell-targeting moiety and an RNAi construct. The invention further provides methods of making the delivery system, and their uses, such as treating phagocytic cell-associated disease conditions.01-19-2012
20120016009Optimized Methods For Delivery Of DSRNA Targeting The PCSK9 Gene - This invention relates to optimized methods for treating diseases caused by PCSK9 gene expression.01-19-2012
20120016007SMALL INTERFERENCE RNA COMPLEX WITH INCREASED INTRACELLULAR TRANSMISSION CAPACITY - A multiplex siRNA complex and a multifunctional nucleic acid structure complex, which have enhanced intracellular delivery capacity are provided. The siRNA complex and the multifunctional nucleic acid structure complex have a novel structure which can be chemically synthesized in an easy manner for a conventional shRNA system for inhibiting the expression of a plurality of genes, while they can inhibit the expression of a plurality of genes at the same time at increased efficiency compared to the conventional siRNA. Also, they have high intracellular delivery capacity and can specifically inhibit the expression of target genes without causing a nonspecific antiviral response, and thus are highly useful as siRNA mechanism-mediated therapeutic agents for treating cancer or viral infection. In addition, the multifunctional nucleic acid structure complex can comprise, in addition to siRNAs, functional oligonucleotides, such as miRNA, antagomiR, an antisense oligonucleotide, an aptamer and ribozyme, and thus can perform various functions at the same time.01-19-2012
20120016006Compositions And Methods For Increasing Cellular Uptake Of RNAi Via SID-1 - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Systemic RNA Interference Defective-1 (SID-I) gene, and methods of using the dsRNA to inhibit expression of SID-1.01-19-2012
20120016004COMPOSITIONS FOR INHIBITING GENE EXPRESSION AND USES THEREOF - The inventors have examined the means for providing more efficacious gene expression blocking compounds. The inventors have discovered new structural features that surprisingly improve the efficacy of gene expression blocking molecules. These features include the presence of multiple 3′ ends and a linker at the 5′ ends. Surprisingly, these features improve the efficacy of the gene expression blocking compounds in a manner that decreases the compound's biologic instability. Even more surprisingly, this effect has been found to be applicable to both DNA and RNA oligonucleotide-based compounds and to have application in traditional antisense and RNAi technologies.01-19-2012
20120016011RNA Interference Mediated Inhibition of Connective Tissue Growth Factor (CTGF) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of CTGF gene expression and/or activity, and/or modulate a CTGF gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against CTGF gene expression.01-19-2012
20120016008ISOLATED GENOMIC POLYNUCLEOTIDE FRAGMENTS FROM THE p15 REGION OF CHROMOSOME 11 ENCODING HUMAN RIBOSOMAL PROTEIN L26 (RIBO26) - Provided herein are isolated genomic polynucleotide fragments from the from the p15 region of chromosome 11 encoding human ribosomal protein L26 (RIBO26) and methods of use.01-19-2012
20120208863MODULATION OF GLUT4 GENE PROMOTER ACTIVITY BY AHNAK - Agents capable of alleviating AHNAK phosphoprotein-mediated repression of GLUT4 gene expression are useful for prevention, treatment, and/or alleviation of insulin resistance associated with obesity, lipotoxicity, hypertension, metabolic syndrome and type 2 diabetes. Preferred agents are double-stranded siRNAs.08-16-2012
20120208860COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF FACTOR VII GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor VII gene.08-16-2012
20120022138MEANS FOR INHIBITING THE EXPRESSION OF ANG2 - The present invention is related to an siRNA comprising an antisense strand and a sense strand, wherein all or a portion of said antisense strand comprises an antisense duplex region, wherein all or a portion of said sense strand comprises a sense duplex region, wherein said antisense duplex region is at least partially complementary to said sense duplex region, wherein said siRNA comprises a duplex region consisting of said antisense duplex region and said sense duplex region, and wherein: a) said antisense strand comprises a nucleotide sequence of SEQ ID NO: 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, 26, 28, 30, 32, 34, 36, 38, 40, 42, 44, 46, 48, 50, 52, 54, 56, 58, 60, 62, 64, 68, 74, 76, 78, 80, 82, 84, 86, 88, 90, 92, 94, 96, 98, 100, 102 or 104; or b) said antisense strand comprises an antisense duplex region, all or a portion of which, is complementary to a portion of SEQ ID NO: 1 or 70.01-26-2012
20120022141COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF A GENE FROM THE JC VIRUS - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a gene from the JC Virus. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by JC virus expression and the expression of a gene from the JC Virus using the pharmaceutical composition; and methods for inhibiting the expression of a gene from the JC Virus in a cell.01-26-2012
20120022142RNA Interference Mediated Inhibition of Signal Transducer and Activator of Transcription 1 (STAT1) Gene Expression Using Short Interfering Nucleic Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of STAT1 gene expression and/or activity, and/or modulate a STAT1 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against STAT1 gene expression.01-26-2012
20120022139Short Interfering Ribonucleic Acid (siRNA) for Oral Administration - Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.01-26-2012
20120022130METHOD OF REGULATING NFATc2 ACTIVITY IN LYMPHOCYTES - A method of decreasing NFATc2 activity in a lymphocyte includes administering to the lymphocyte an amount of an NFATc2 mRNA antagonist that binds to a binding site on the 3′UTR of NFATc2 mRNA effective to decrease the activity of NFATc2 mRNA in the lymphocyte.01-26-2012
20120022137METHOD OF CONTROLLING INITIAL DRUG RELEASE OF siRNA FROM SUSTAINED-RELEASE IMPLANTS - The present invention provides an intraocular implant comprising siRNA combined with a excipient effective to retard the initial release of the siRNA from an implant, wherein said siRNA and excipient is associated with a biocompatible polymer (e.g., a polymeric matrix), configured to release said siRNA into the eye of a patient at therapeutic levels for a time sufficient to treat an ocular condition or disease.01-26-2012
20120022133ANTISENSE MODULATION OF CHOLESTERYL ESTER TRANSFER PROTEIN EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of cholesteryl ester transfer protein. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding cholesteryl ester transfer protein. Methods of using these compounds for modulation of cholesteryl ester transfer protein expression and for treatment of diseases associated with expression of cholesteryl ester transfer protein are provided.01-26-2012
20120022136Cellular Genes Regulated by HIV-1 Infection and Methods of Use Thereof - This invention provides cellular gene products which have anti-apoptotic activity in HIV-1 infected cells and provides agents for the inhibition of the cellular gene products.01-26-2012
20120022132COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF MUTANT EGFR GENE - The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a mutant Epidermal Growth Factor Receptor (EGFR), and methods of using the dsRNA to inhibit expression of mutant EGFR.01-26-2012
20120022131BREAST CANCER RELATED GENE RQCD1 - The present invention provides methods for detecting and diagnosing cancer, such methods involving the determination of the expression level of the RQCD1, GIGYF1 or GIGYF2 genes. These genes were discovered to discriminate cancer cells from normal cells. Furthermore, the present invention provides methods of screening for therapeutic agents useful in the treatment of cancer and methods for treating cancer. Moreover, the present invention provides siRNAs targeting the RQCD1, GIGYF1 and/or GIGYF2 genes, all of which are suggested to be useful in the treatment of cancer.01-26-2012
20120022129Targeting of Histone Deacetylase 2, Protein Kinase CK2, and Nuclear Factor NRF2 For Treatment of Inflammatory Diseases - Methods for the treatment or prevention of diseases which are caused by the degradation of histone deacetylase 2 (HDAC2) in cells are described. The diseases which may be treated by the methods of the invention include chronic obstructive pulmonary disease (COPD) and asthma. The invention provides methods for treating or preventing of diseases caused by the degradation of HDAC2 by providing to the subject in need of treatment or prevention a molecular compound capable of preventing the degradation of HDAC2. Such molecular compounds include protein kinase CK2 inhibitors, ubiquitination inhibitors, ubiquitin-proteosome inhibitors, nuclear factor (erythroid-derived 2)-like 2 (Nrf2) activators and MAPK phosphatase 1 activators. Methods are also provided for the treatment and prevention of diseases caused by the degredation of HDAC2 by interfering with the expression of protein kinase CK2 or by increasing expression of Nrf2.01-26-2012
20120022128PKIB and NAALADL2 for Target Genes of Prostate Cancer Therapy and Diagnosis - The invention features methods for detecting prostate cancer, especially hormone-refractory prostate cancer (HRPC) or castration-resistant prostate cancer (CRPC), by detecting over-expression of PKIB or NAALADL2 compared the normal organs. Also disclosed are methods of identifying compounds for treating and preventing prostate cancer including HRPC, based on the over-expression of PKIB or NAALADL2 in the prostate cancer, the cell proliferation function of PKIB or NAALADL2, the intracellular localization of PKIB or NAALADL2 or the interaction between PKIB and PKA-C. Also, provided are a method for treating prostate cancer by administering a double-stranded molecule against the PKIB or NAALADL2 gene. The invention also provides products, including the double-stranded molecules and vectors encoding them, as well as compositions comprising the molecules or vectors, useful in the provided methods.01-26-2012
20120022143RNA Interference Mediated Inhibition of the Thymic Stromal Lymphopoietin (TSLP) Gene Expression Using Short Interfering Nucliec Acid (siNA) - The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of TSLP gene expression and/or activity, and/or modulate a TSLP gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against TSLP gene expression.01-26-2012
20120022145Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.01-26-2012
20120022140Method of treating a cancer with a survivin antisense oligonucleotide and paclitaxel - Provided is a method of treating cancer of the stomach, comprising administering to a patient a therapeutically effective combination of a Survivin antisense oligonucleotide and paclitaxel.01-26-2012
20120022134METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA - The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in said method.01-26-2012
20120059045METHODS OF USING OLIGOMERIC COMPOUNDS COMPRISING 2'-SUBSTITUTED NUCLEOSIDES - The present disclosure provides oligomeric compounds comprising at least one 2′-fluoroethoxy modified nucleoside of formula I and methods of using these oligomeric compounds. The methods provided herein include contacting a cell or administering to an animal at least one of the oligomeric compounds. In certain embodiments, the oligomeric compounds hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.03-08-2012
20120059047DIFFERENTIATION MODULATING AGENTS AND USES THEREFOR - The present invention is directed to methods and agents for modulating the differentiation potential and/or proliferation of preadipocytes. More particularly, the present invention discloses methods and agents for modulating a fibroblast growth factor (FGF) signaling pathway, especially the FGF-1 or FGF-2 signaling pathway, for treating or preventing adiposity-related conditions including, but not limited to, obesity, lipoma, lipomatosis, cachexia or lipodystrophy or the loss of adipose tissue in trauma or atrophic conditions.03-08-2012
20120059046INHIBITION OF MAP4K4 THROUGH RNAI - RNAi constructs directed to MAP4K4 that demonstrate unexpectedly high gene silencing activities, and uses thereof are disclosed. The blunt-ended constructs have a double-stranded region of 19-49 nucleotides. The constructs have selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. For example, the strands may be modified (e.g., one or both ends of the sense strand is modified by 2′-O-methyl groups), such that the construct is not cleaved by Dicer or other RNAse III, the antisense strand may also be modified by a 2′-O-methyl group at the penultimate 5′-end nucleotide to greatly reduce off-target silencing.03-08-2012
20120059048METHODS FOR DIAGNOSING AND TREATING SQUAMOUS CELL CARCINOMA UTILIZING miRNA-205 AND INHIBITORS THEREOF - Disclosed are diagnostic and therapeutic methods related to squamous cell carcinoma. In particular, the diagnostic methods relate to detecting miRNA-205, thereby diagnosing an aggressive form of squamous cell carcinoma. The therapeutic methods relate to inhibiting the function of miRNA-205, thereby treating an aggressive form of squamous cell carcinoma.03-08-2012
20120059044METHODS OF UTILIZING THE ARRESTIN-2/STAM-1 COMPLEX AS A THERAPEUTIC TARGET - Methods of utilizing the arrestin-2/sTAM-1 complex as a therapeutic target. The methods include treating cells of a living organism to mediate an interaction between arrestin-2 and STAM-1 adapter protein molecules, wherein the interaction is characterized by the arrestin-2 adapter protein molecule directly binding to the STAM-2 adapter protein molecule. Pharmacological agents can be identified for therapeutic uses by determining whether the pharmacological agent disrupts the interaction between the arrestin-2 and STAM-1 adapter protein molecules.03-08-2012
20120059042METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS - The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5′-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.03-08-2012
20120059041Methods for Extending Lifespan in Subject - Disclosed is a method for extending lifespan in a subject. By screening for mutations that enhance resistance to multiple stresses, the invention identified multiple alleles of alpha-1, 2-mannosidase I (mas1) which, in addition to promoting stress resistance, also extended longevity. Meanwhile, longevity enhancement of a subject is also observed when either the expression of mas1 or its downstream gene Edm1 is reduced. Furthermore, this invention also found that the down-regulating mas1 and Edm1 may extend longevity by modulating DR (Dietary Restriction). Thus, via molecular biology techniques, the expression of the target genes such as mas1 and Edm1 can be regulated, and the lifespan extension for a subject also can be achieved.03-08-2012
20120065242POLYMERIC NANO-PARTICLES FOR siRNA DELIVERY USING CHARGE INTERACTION AND COVALENT BONDING - Disclosed is a polymer-siRNA delivery carrier in which a siRNA is combined with a polymer and the use thereof. More specifically, there is disclosed a stable in vivo polymer-siRNA delivery carrier in which a polymer and a siRNA are combined by using charge interaction and biodegradable covalent bonding at the same time and the use thereof.03-15-2012
20100063133ANTISENSE ANTIVIRAL COMPOUND AND METHOD FOR TREATING ARENAVIRUS INFECTION - The invention provides antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Arenaviridae family and in the treatment of a viral infection. The compounds are particularly useful in the treatment of Arenavirus infection in a mammal. The antisense antiviral compounds are substantially uncharged morpholino oligonucleotides have a sequence of 12-40 subunits, including at least 12 subunits having a targeting sequence that is complementary to a region associated with viral RNA sequences within a 19 nucleotide region of the 5′-terminal regions of the viral RNA, viral complementary RNA and/or mRNA identified by SEQ ID NO:1.03-11-2010
20120029058Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.02-02-2012
20120157512Preventing and Curing Beneficial Insect Diseases Via Plant Transcribed Molecules - Methods and compositions for transforming plants to express polynucleotides capable of gene silencing gene expression in pathogens of beneficial insets such as IAPV, 06-21-2012
201201575115' PHOSPHATE MIMICS - The present invention provides nucleosides and oligonucleotides comprising a 5′ phosphate mimics of formula (IVc) or (Vc). One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such dinucleotide of formula (Ia). Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.06-21-2012
20120065250siRNA Targeting Apolipoprotein B (APOB) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for APOB.03-15-2012
20120065247MODULATING IRES-MEDIATED TRANSLATION - Provided herein are compounds and methods for use in preventing or treating a viral infection mediated by a virus comprising an IRES-containing RNA molecule or cancer related to an increase or decrease in IRES-mediated translation of an RNA molecule. Also provided are methods of inhibiting or promoting IRES-mediated translation. Also provided are methods of screening for an agent that inhibits IRES-mediated translation. 03-15-2012
20120208865EXON SKIPPING THERAPY FOR DYSFERLINOPATHIES - The present invention relates to methods for restoring the function of a mutated dysferlin comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction. Particularly, the splicing of exon 32 is prevented. The present invention also relates to a method for treating a dysferlinopathy in a patient in need thereof, comprising the step of administering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of one or more exons which encode amino acid sequences that cause said dysfunction. Particularly, the splicing of exon 32 is prevented.08-16-2012
20120122955USE OF LNA APOB ANTISENSE OLIGOMERS FOR THE TREATMENT OF ACUTE CORONARY SYNDROMES - The present invention relates to the use of LNA antisense apoB oligonucleotides for the treatment of acute coronary syndrome.05-17-2012
20120071541Micro-RNA Associated With Rheumatoid Arthritis - An object of the present invention is to provide a novel marker for rheumatoid arthritis (RA), and more specifically, to provide a marker whose expression may be specifically increased or decreased in RA. Another object of the present invention is to confirm whether or not miRNA serving as the marker is involved as the etiology of RA, and to provide an inspection method for RA and a therapeutic agent for RA each using the miRNA involved. The marker includes miRNA (for example, miR12403-22-2012
20120071540Compositions and Methods Using siRNA Molecules and siRNA Cocktails for the Treatment of Breast Cancer - The present invention provides small interfering RNA (siRNA) molecules, compositions containing the molecules, and methods of using the molecules and compositions to treat breast cancer. In one aspect, a multi-targeted siRNAi cocktail is disclosed. The siRNA molecules may be encapsulated in nanoparticles to further enhance their anti-cancer activity. The compositions may also be used in combination with other anti-cancer agents, such as bevacizumab.03-22-2012
20120071539COMPOUNDS AND METHODS FOR MODULATING THE SILENCING OF A POLYNUCLEOTIDE OF INTEREST - Methods and compositions comprising chemical compounds that modulate the silencing of a polynucleotide of interest in a cell are provided. Such chemical compounds when used in combination with an appropriate silencing element can be used to modulate (increase or decrease) the level of the polynucleotide targeted by the silencing element. Methods of using such compositions both in therapies involving RNAi-mediated suppression of gene expression, as well as, in vitro methods that allow for the targeted modulation of expression of a polynucleotide of interest are provided. Pharmaceutical or cosmetic compositions comprising such compounds and silencing elements also are disclosed. Methods for screening a compound of interest for the ability to modulate the activity of a heterologous silencing element also are provided.03-22-2012
20110092574mRNA Cap Analogs - Dinucleotide cap analogs are disclosed, modified at different phosphate positions with a boranophosphate group or a phosphoroselenoate group. The analogs are useful as reagents in the preparation of capped mRNAs and have increased stability both in vitro and in vivo. They may be used as inhibitors of cap-dependent translation. Optionally, the boranophosphate or phosphoroselenoate group has a 2′-O or 3′-O-alkyl group, preferably a methyl group, producing analogs called BH04-21-2011
20110092573MODIFICATION OF MYD88 SPLICING USING MODIFIED OLIGONUCLEOTIDES - Antisense compounds, compositions and methods are provided for modulating the expression of MyD88. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding MyD88. Methods of using these compounds for modulation of MyD88 expression and for treatment of diseases associated with expression of MyD88 are provided.04-21-2011
20110092572MODULATION OF GROWTH HORMONE RECEPTOR EXPRESSION AND INSULIN-LIKE GROWTH FACTOR EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of growth hormone receptor and/or insulin like growth factor-I (IGF-I). The compositions comprise oligonucleotides, targeted to nucleic acid encoding growth hormone receptor. Methods of using these compounds for modulation of growth hormone receptor expression and for diagnosis and treatment of disease associated with expression of growth hormone receptor and/or insulin-like growth factor-I are provided. Diagnostic methods and kits are also provided.04-21-2011
20110092571COMPOSITIONS AND METHODS FOR SIRNA INHIBITION OF ICAM-1 - RNA interference using small interfering RNAs which are specific for the ICAM-1 gene inhibits expression of this gene. Diseases which involve ICAM-1-mediated cell adhesion, such as inflammatory and autoimmune diseases, diabetic retinopathy and other complications arising from type I diabetes, age related macular degeneration and many types of cancer, can be treated by administering the small interfering RNAs.04-21-2011
20110092570OLIGOMERIC COMPOUNDS COMPRISING TRICYCLIC NUCELOSIDES AND METHODS FOR THEIR USE - The present disclosure provides tricyclic nucleosides, oligomeric compounds comprising at least one of the tricyclic nucleosides and methods of using the oligomeric compounds. The methods provided herein include contacting a cell or administering to an animal at least one of the oligomeric compounds. In certain embodiments, the oligomeric compounds hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.04-21-2011
20110092569Administration of Exogenous mi/siRNA - Certain genes controlled by endogenous miRNA are actually upregulated upon the transfection of exogenous mi/siRNA. Based on this, methods of determining whether administration of mi/siRNA will have a deleterious effect by upregulating certain genes are provided. Comparison of sequences of exogenous mi/siRNA allows selection of exogenous miRNA to be administered to cell to enhance or limit this affect, and therefore to control unwanted disregulation, or to upregulate an endogenous miRNA-regulated gene of interest.04-21-2011
20110092568MODULATION OF MLCK-L EXPRESSION AND USES THEREOF - In various aspects and embodiments the invention provides methods and reagents for controlling gene expression, and for treating disorders and diseases. Embodiments provide methods and reagents specifically for the regulation of MLCK expression and for the use thereof in treating disorders and diseases. Various embodiments provide methods and reagents for specifically down regulating the expression of MLCK-L more efficiently than that of MLCK-S, and for the use thereof in treating disorders and diseases. Embodiments provide siNA for the same, particularly siRNAs. Various of the embodiments are useful for the treatment of inflammatory disorders and diseases, including, for one example in the regard, Asthma.04-21-2011
20110092567Cannabinoid 2 (Cb2) Receptor Gene Promoter and Unique RNA Transcripts in B Cells and Methods of Use - Cannabinoid receptor 2 (CB04-21-2011
20110092566Treatment of cancer with aldose reductase inhibitors - Provided herein are methods of treating a pathophysiological state or symptoms thereof resulting from aldose reductase-mediated signaling in a cytotoxic pathway in a subject using an inhibitor of aldose reductase. Particularly, specific inhibitors may be small molecules such as fidarestat or siRNA. Also, methods of treating breast and prostate cancers or suppressing metastasis of colon cancer thereof using the siRNAs and aldose reductase inhibitors are provided.04-21-2011
20110092565METHOD OF TREATING NEURODEGENERATIVE DISEASE - Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.04-21-2011
20120115934Organic Compositions to Treat Beta-ENaC-Related Diseases - The present disclosure relates to RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.05-10-2012
20120115933Organic Compositions to Treat Beta-ENaC-Related Diseases - The present disclosure relates to RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.05-10-2012
20120115929Means And Methods For Counteracting, Preventing And/Or Determining Heart Failure, Or A Risk Of Heart Failure - The present invention relates to a method for diagnosing and/or prognosing heart failure in a subject the method comprising the step of determining the level of at least one genetic marker in a body fluid sample obtained from said subject, wherein said at least one genetic marker is an mi RNA. The invention further relates to a method for treating or preventing heart failure in a subject the method comprising the step of decreasing within said subject the expression, amount, and/or activity of at least one mi RNA, or decreasing within said subject the interaction of at least one mi RNA with its m RNA target or increasing within said subject the expression, amount, and/or activity of the m RNA target of at least one mi RNA.05-10-2012
20120115927PHARMACEUTICAL AGENT FOR PREVENTING CELL DEATH - Disclosed is a pharmaceutical agent for preventing cell death. The occurrence of cell death can be prevented by inhibiting the function of Int6 protein in an affected area. Then, a pharmaceutical agent comprising a substance capable of inhibiting the function of Int6 protein is prepared. The pharmaceutical agent can be used for preventing cell death.05-10-2012
20120115925Allelic Variants Associated with Advanced Age-Related Macular Degeneration - The invention relates to allelic variants and haplotypes of the Complement Factor H (CFH) gene, the Complement Component 3 (C3) gene, and the Complement Factor D (CFD) gene, associated with either an elevated or a reduced risk that an individual will develop age-related macular degeneration (AMD), methods of diagnosing such risk in an individual based on the presence or absence of such variants, and methods and reagents for diagnosis and treatment of AMD.05-10-2012
20100184824RNA Interference Mediated Inhibition of Interleukin and Interleukin Receptor Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of interleukin and/or interleukin receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of interleukin and/or interleukin receptor genes.07-22-2010
20090209622Diagnosis of anxiety disorders - The invention relates to methods for diagnosing a genetic predisposition or susceptibility for anxiety disorders in a human. The methods include detecting particular markers at the human RGS2 locus in a sample obtained from the human. The invention also relates to the improved diagnosis that is based on the analysis of haplotypes for the RGS2 locus.08-20-2009
20100160413Double-stranded ribonucleic acids with rugged physico-chemical structure and highly specific biologic activity - The invention relates to our discovery of a novel double-stranded ribonucleic acid (dsRNA) having specific biological activities, which includes acting as a selective agonist for activation of the Toll-like receptor 3. Its “rugged” molecular structure as measured by physico-chemical techniques is resistant to molecular unfolding (i.e., denaturation). This structure appears to be responsible for increased efficacy of dsRNA in therapeutic applications and improved biological activity (e.g., used as an immunoregulatory agent). Medicaments, processes for their manufacture, and methods for their use are provided herein.06-24-2010
20100324118Method for the Promotion of Angiogenesis, Vascularization, or Vascular Repair or for the Inhibition of Tumor Angiogenesis - The invention relates to a method for influencing the miR-92 expression in a cell, comprising the following steps: (a) providing a cell; and (b1) reducing the miR-92 expression in the cell in order to promote the vascularization or vessel repair by introducing an antisense molecule against miR-92 into the cell, or (b2) increasing the miR-92 expression in the cell for an inhibition of the tumor angiogenesis by introducing a construct into the cell, wherein said construct includes an expressible miR-92 sequence. Furthermore, the invention relates to a pharmaceutical composition, comprising an agent for reducing the miR-92 activity or expression in a cell in the form of an antisense molecule against miR-92, or an agent for increasing the miR-92 expression in a cell in the form of a construct for expressing miR-92.12-23-2010
20120108652OLIGOMERS - Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.05-03-2012
20100160414Treating Picornavirus Infection by Targeting MicroRNA miR-141 - Treatment of picornavirus infection by inhibiting miR-141 activity. Also disclosed herein are a method for identify miR-141 inhibitory compounds and a method for identifying a target viral infection to be treated by anti-miR-141 therapy.06-24-2010
20120157510Controlling The Potential Of Primate Neural Stem Cells By Regulating Pax6 - A transcription factor both necessary and sufficient for human neuroectoderm specification, Pax6, as well as applications thereof, is disclosed.06-21-2012
20120309814TREATMENT OF PYRROLINE-5-CARBOXYLATE REDUCTASE 1 (PYCR1) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO PYCR1 - The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Pyrroline-12-06-2012
20120309813DELIVERY OF dsRNA TO ARTHROPODS - The invention is to methods of gene silencing in arthropods using dsRNA. The method is include contacting the arthropod with, and/or directly feeding the arthropod, the dsRNA to the arthropods to deliver the dsRNA to arthropod tissues. It is envisaged that the methods of the invention will have use in determining the biological function of genes in arthropods. Methods of pest control of arthropods, and of protecting arthropods against parasites and predators are provided. Transgenic arthropods expressing dsRNA molecules are also provided by the present invention.12-06-2012
20100197762ANTISENSE COMPOUNDS - Provided herein are gapmer oligomeric compounds for reduction of target RNA in vivo comprising different nucleotide modifications within one or both wing regions. Also provided are methods of using such oligomeric compounds, including use in animals. In certain embodiments, such compound have desirable potency and toxicity characteristics.08-05-2010
20120077860Adeno-Associated Viral Vector for Exon Skipping in a Gene Encoding a Dispensable Domain Protein - The invention concerns an adeno-associated viral vector comprising: 03-29-2012
20110065777DOUBLE-STRANDED RNA (dsRNA) AND METHOD OF USE FOR INHIBITING EXPRESSION OF A FUSION GENE - Specific inhibition of expression of a fusion gene in mammals occurs using a short, double-stranded ribonucleic acid molecule (dsRNA). The dsRNA comprises two separate non-linked RNA strands, an 51 strand and a complementary strand. The strands are 20 to 23 nucleotides in length, and the 51 strand is complementary to the fusion junction of the AML-1/MTG-8 fusion gene. The dsRNA also comprises at least 3 nucleotides on each side of the fusion junction. The dsRNA can be introduced into and maintained in a mammalian cell under conditions and for a time sufficient to obtain degradation of mRNA of the fusion gene to inhibit expression of the fusion gene. The dsRNAs and methods described are useful for treating diseases caused by chromosomal aberrations, particularly malignant diseases such as lymphoma and leukemia.03-17-2011
20110065776Method for Treating Hepatitis C Infection - The present invention relates to a method for treating hepatitis C virus (HCV) infection, comprising administrating a subject in need thereof with a therapeutically effective amount of an inhibitor against a serine/threonine kinase (AKT) and an activator thereof. A method for screening a candidate agent for treating hepatitis C infection determined by the presence of an inhibition of AKT function is also provided.03-17-2011
20110065775COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF SGLT2 - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.03-17-2011
20110065772TREATMENT OF RHEUMATOID ARTHRITIS - The present invention provides a method for treating or inhibiting rheumatoid arthritis in a subject, the method c