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800 - Multicellular living organisms and unmodified parts thereof and related processes


800013000 - Transgenic nonhuman animal (e.g., mollusks, etc.)

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Class / Patent application numberDescriptionNumber of patent applications / Date published
800018000 Mouse 159
800015000 Bovine 31
800017000 Swine 16
800016000 Sheep 5
20110023159OVINE GENOME EDITING WITH ZINC FINGER NUCLEASES - The present invention provides a genetically modified ovine or cell comprising at least one edited chromosomal sequence. In particular, the chromosomal sequence is edited using a zinc finger nuclease-mediated editing process. The disclosure also provides zinc finger nucleases that target specific chromosomal sequences in the ovine genome.01-27-2011
20100088778Methods of Treatment, and Diagnosis of Epilepsy by Detecting Mutations in the SCN1A Gene - A method for the diagnosis of an epilepsy syndrome, including SMEI or an SMEI-related syndrome, in a patient comprising testing for an alteration in the SCN1A gene in a sample obtained from the patient; and if an alteration is identified, comparing said alteration to any one of those listed in Table 3, wherein if said alteration is identical to any one of those listed in Table 3, a diagnosis of an epilepsy syndrome, including SMEI or an SMEI-related syndrome, in said patient is made in accordance with the correlation set forth in Table 3.04-08-2010
20090077679GENE SILENCING BY SYSTEMIC RNA INTERFERENCE - Nucleic acid and protein sequences relating to a gene required for systemic RNAi are disclosed. The SID-1 protein is shown to be required for systemic RNAi. Nucleic acids, vectors, transformed cells, transgenic animals, polypeptides, and antibodies relating to the sid-1 gene and protein are disclosed. Also provided are methods for reducing the expression of a target gene in a cell, a population of cells, or an animal.03-19-2009
20100077496MO-1, A Gene Associated With Morbid Obesity - MO-1 is a newly identified gene and gene product associated with morbid obesity. Isolated MO-1 nucleic acids, MO-1 polypeptides, oligonucleotides that hybridize to MO-1 nucleic adds, and vectors, including expression vectors, comprising MO-1 nucleic acids are disclosed, as are isolated host cells, antibodies, transgenic non-human animals, compositions, and kits relating to MO-1. Methods of detecting the presence of MO-1 nucleic acid, screening for agents which affect MO-1 activity, and screening for MO-1 variants are also disclosed.03-25-2010
20130024960OPTIMISED CODING SEQUENCE AND PROMOTER - An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.01-24-2013
20110179508ENTEROVIRUS TYPE 71 PROTEIN AND METHOD FOR PRODUCTION - The invention provides a new type of a capsid protein VP1 of human enterovirus 71, named as MEL701-VP1 and functional/structural variants thereof, which is used for protection against enterovirus. The transgenic animal producing the protein, the composition comprising the protein and the method for production thereof are also provided.07-21-2011
20090217400Enzymes, cells and methods for site specific recombination at asymmetric sites - The present invention relates to enzymes, compositions and methods for catalyzing asymmetric recombination of non-palindromic recombination sites in a cell free system, in isolated cells or in living organisms. The enzymes and methods of the invention are suitable for mediating specific recombinations between DNA sequences comprising specific recombination sites without being limited to strict palindromic symmetry within each recombination site.08-27-2009
20110191874COMPOSITIONS AND METHODS FOR DETECTING AND MODULATING CELL DEATH BY A TRANSLATION REGULATED GENE EXPRESSION SYSTEM - The technology relates to a nucleic acid expression cassette comprising a TR element encoding an mRNA molecule that is translated in stressed and/or dying cells, and a nucleotide sequence operably linked to the TR element, that is a first open reading frame (ORF) sequence and encodes a polypeptide or a fragment thereof and is co-translated with the TR element. The technology further relates to mammalian cells and a transgenic animal comprising such expression cassette. Further included are kits comprising the expression cassette, and methods for determining toxicity, and killing a target cell08-04-2011
20090193531Methods and compositions for RNA Interference - The present invention provides methods for attenuating gene expression in a cell using gene-targeted double stranded RNA (dsRNA). The dsRNA contains a nucleotide sequence that hybridizes under physiologic conditions of the cell to the nucleotide sequence of at least a portion of the gene to be inhibited (the “target” gene).07-30-2009
20090019560CLONED RAT BY NUCLEAR TRANSFER USING ENUCLEATED RAT OOCYTES - The invention relates to a method for cloning in the rat by nuclear transfer. The invention further relates to the rats obtained thus, in the foetal or adult state, as well as use thereof for the production of molecules of interest or as study models.01-15-2009
20130042334Capping-Prone RNA Polymerase Enzymes and Their Applications - The invention provides a chimeric enzyme comprising at least one catalytic domain of a RNA triphosphatase, at least one catalytic domain of a guanylyltransferase, at least one catalytic domain of a N02-14-2013
20100325746Methods and sequences to suppress primate huntington gene expression in vivo - Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including 12-23-2010
20100107264DEVELOPMENT OF INFLUENZA A ANTIVIRALS - The present invention includes compositions, methods and systems to isolate and characterize novel antiviral agents by contacting the antiviral agent with the F2F3 zinc fingers of a CPSF30 protein and an Influenza A NS1A protein; and determining whether the binding between the CPSF30 protein and the Influenza A NS1A protein is reduced.04-29-2010
20120167238UNIVERSAL PEPTIDE TAGS FOR TRANSGENE POLYPEPTIDE ANALYSIS BY MASS SPECTROMETRY - Compositions and methods that allow for the rapid detection and accurate quantification of any polypeptides of interest are provided. Compositions include isolated polypeptides comprising at least one universal peptide tag, as well as isolated polynucleotides encoding such polypeptides. The universal peptide tags can be quantified by methods including, but not limited to, mass spectrometry, and can act as surrogates for determining the concentration of the polypeptides comprising the universal peptide tags. Methods provide for the detection and/or quantification of any polypeptides of interest that comprise at least one universal peptide tag, including methods using mass spectroscopy techniques. Methods are also provided for producing hosts, or cells or parts thereof, that comprise polypeptides comprising at least one universal peptide tag. Hosts, or cells, or parts thereof, include mammalian, bacterial, insect, yeast, viral or plant.06-28-2012
20090307790ISOLATION OF THE T-COMPLEX DISTORTERS AND APPLICATIONS THEREOF - The present invention relates to a method for producing a transgenic non human male animal, preferably a mammal, fish, bird or insect, wherein the transgene(s) confer(s) a change in the transmission ratio of (a) genetic trait(s) to the offspring of said non human male animal, preferably mammal, fish, bird or insect to a non-Mendelian ratio, said method comprising introducing (a) a first nucleic acid molecule encoding an expression product with a Responder function into a chromosome of a non-human germ cell, (fertilized) egg cell, embryonic cell or a cell derived therefrom, of the same species as the transgenic male to be prepared, said chromosome containing or conferring said genetic trait(s), thereby linking on said chromosome said Responder function to the genetic trait(s); and (b) at least one second nucleic acid molecule encoding an expression product with a Distorter function into (a) chromosome(s) of a non-human germ cell, (fertilized) egg cell, embryonic cell or a cell derived therefrom, of the same species as the transgenic male to be prepared, wherein said expression product with a Distorter function is a factor involved in G protein signaling, wherein said first nucleic acid molecule encoding an expression product with Responder function and said at least one second nucleic acid molecule encoding an expression product with a Distorter function are introduced into the same or different chromosomes.12-10-2009
20120192298METHOD FOR GENOME EDITING - The present invention encompasses a method for creating an animal or cell with at least one chromosomal edit. In particular, the invention relates to the use of targeted zinc finger nucleases to edit chromosomal sequences. The invention further encompasses an animal or a cell created by a method of the invention.07-26-2012
20090094709Use of protein phosphatase 2Ce (PP2Ce) having dephosphorylating action on AMPK - A drug includes, RNA interference with protein phosphatase 2Cε (PP2Cε) as an active ingredient. According to the present invention, the activation and deactivation of AMPK can be regulated.04-09-2009
20110023157EQUINE GENOME EDITING WITH ZINC FINGER NUCLEASES - The present invention provides a genetically modified equine or cell comprising at least one edited chromosomal sequence. In particular, the chromosomal sequence is edited using a zinc finger nuclease-mediated editing process. The disclosure also provides zinc finger nucleases that target specific chromosomal sequences in the equine genome.01-27-2011
20130067609Mammalian Genes Involved in Tularemia and Other Infections - The present invention relates to nucleic acid sequences and cellular proteins encoded by these sequences that are involved in infection or are otherwise associated with the life cycle of one or more pathogens.03-14-2013
20110023156FELINE GENOME EDITING WITH ZINC FINGER NUCLEASES - The present invention provides a genetically modified feline or cell comprising at least one edited chromosomal sequence. In particular, the chromosomal sequence is edited using a zinc finger nuclease-mediated editing process. The disclosure also provides zinc finger nucleases that target specific chromosomal sequences in the feline genome.01-27-2011
20110283374METHOD FOR PRODUCING HETEROGENOUS EMBRYONIC CHIMERIC ANIMAL USING A STEM CELL - A heterologous chimeric animal can be produced by a method comprising the steps of: (A) injecting a stem cell into a blastocyst cavity in a blastocyst stage of an animal heterologous to that of the stem cell, or mixing the stem cell with a divided fertilized egg of the animal heterologous to that of the stem cell; and (B) growing a cell mass including the stem cell prepared in the step (A) into a chimeric animal between a species of the stem cell and a species of the heterologous animal.11-17-2011
20110302666PRODUCTION AND USE OF RAT SPERMATOGONIAL STEM CELL LINES - A spermatogonial stem cell line that is derived from testes rats characterized by a desirable genetic background can serve as a source for cells to transplant into male-sterile recipient animals that are immuno-compatible with the spermatogonial line. Rat cells thus transplanted readily develop into fertilization-competent, haploid male gametes, with little or no endogenous sperm competition generated by the testes of the male-sterile recipient. This approach, constituting the first vector system for the use of rat spermatogonial lines from in vitro culture in generating mutant rats on a desired genetic background, effects maximal germline transmission of donor haplotypes from the transplanted spermatogonial cells.12-08-2011
20100077495COMPOSITIONS AND METHODS FOR THE EXPRESSION OF NUCLEIC ACIDS - Compositions and methods are provided herein for the expression of nucleic acids. Compositions and methods are also provided herein for inducible expression of nucleic acids in transgenic cells and animals using transposon-based nucleic acid constructs. Compositions and methods are also provided herein for modulation of endogenous gene expression.03-25-2010
20090178152Use of the Ornithine Transcarbamylase (OTC), as a Marker for Diagnosing Brain Damages - The present invention pertains to the domain of brain diseases, and provides novel markers and methods for diagnosing a brain alteration in an individual, especially in patients suffering from neurodegenerative diseases such as Alzheimer's disease. The present invention also provides tools for evaluating the probability, for an individual, of developing the disease, as well as a target for identifying new drugs for treating neurodegenerative diseases such as Alzheimer's disease. In particular, the invention provides a genetic marker based on combination of two single nucleotide polymorphism, at positions −389 and −241 of the ornithine transcarbamylase (OTC) gene.07-09-2009
20120110686Cand45 tRNA-Derived Expression System for Gene Modulation - The present invention relates to systems and methods for modulating gene expression and applications thereof. Provided is a novel expression system to generate RNaseZ and RNA polymerase III dependent RNAs to regulate genes and control the timing and the location of the regulation by supplying synthetic or expressed oligonucleotide antisense to a small RNA.05-03-2012
20100122363Certain human genomic DNA associated with total red-green colorblindness - Disclosed is human genomic DNA having the following STR marker profile:05-13-2010
20100122362CELLS EXPRESSING ALPHA-SYNUCLEIN AND USES THEREFOR - Disclosed are mammalian neuronal cells expressing alpha synuclein as well as methods of screening to identify compounds that decrease toxicity induced by alpha synuclein expression. Compounds identified by such screens can be used to treat or prevent synucleinopathies such as Parkinson's disease, familial Parkinson's disease, Lewy body disease, the Lewy body variant of Alzheimer's disease, dementia with Lewy bodies, multiple system atrophy, or the Parkinsonism-dementia complex of Guam.05-13-2010
20090255004G-Protein Coupled Receptor and Uses Therefor - The present invention is based on the identification of a G-protein coupled receptor (GPCR) that is expressed predominantly in the brain and placenta and nucleic acid molecules that encoded the GPCR, which is referred to herein as the hCAR protein and hCAR gene respectively (for human Constitutively Active Receptor). Based on this identification, the present invention provides: (1) isolated hCAR protein; (2) isolated nucleic acid molecules that encode an hCAR protein; (3) antibodies that selectively bind to the hCAR protein; (4) methods of isolating allelic variants of the hCAR protein and gene; (5) methods of identifying cells and tissues that express the hCAR protein/gene; (6) methods of identifying agents and cellular compounds that bind to the hCAR protein; (7) methods of identifying agents that modulate the expression of the hCAR gene; and (8) methods of modulating the activity of the hCAR protein in a cell or organism.10-08-2009
20090307791CALCIUM-ACTIVATED CHLORIDE CHANNEL AND METHODS OF USE THEREOF - The present invention provides a cloned, isolated calcium-activated chloride channel, and a polynucleotide comprising a nucleotide sequence encoding the channel. The present invention further provides a genetically modified cell comprising a subject polynucleotide, and use of the cells to identify agents that modulate calcium-activated chloride channel activity. The present invention further provides genetically modified cells and non-human animals that do not express a subject calcium-activated chloride channel.12-10-2009
20090113563Probe for detecting nuclear receptor agonist or antagonist and method for screening agonist or antagonist to nuclear receptor with the use of the same - A probe for detecting an agonist or an antagonist to a nuclear receptor, in which, at least, a ligand-recognition site containing a ligand-binding domain of the nuclear receptor is connected with a binding-responsive site containing a peptide chain that specifically binds to a coactivator-binding site in the ligand-binding domain by a flexible linker to construct a fusion structure [ligand-recognition site/linker/binding-responsive site], and two reporters are connected with the respective ends of the fusion structure.04-30-2009
20100287635Cloned Canines and Method for Producing Thereof - Disclosed herein are a cloned canine and a production method thereof. The method comprises the steps of enucleating the oocyte of a canine to prepare an enucleated recipient oocyte, conducting nuclear transfer into the enucleated oocyte using a canine somatic cell as a nuclear donor cell under optimized conditions so as to prepare a nuclear transfer embryo, and transferring the nuclear transfer embryo into the oviduct of a surrogate mother. The present invention provides a method for producing cloned canines and thus, can contribute to the development of studies in veterinary medicine, anthropology and medical science such as the propagation of superior canines, the conservation of rare or nearly extinct canines, xenotransplantation and disease animal models.11-11-2010
20090265798Desaturase genes and uses thereof - The subject invention relates to the identification of genes involved in the desaturation of polyunsaturated fatty acids at carbon 5 (i.e., “Δ5-desaturase”) and at carbon 6 (i.e., “Δ6-desaturase”) and to uses thereof. In particular, Δ5-desaturase may be utilized, for example, in the conversion of dihomo-γ-linolenic acid (DGLA) to arachidonic acid (AA) and in the conversion of 20:4n-3 to eicosapentaenoic acid (EPA). Delta-6 desaturase may be used, for example, in the conversion of linoleic (LA) to γ-linolenic acid (GLA). AA or polyunsaturated fatty acids produced therefrom may be added to pharmaceutical compositions, nutritional compositions, animal feeds, as well as other products such as cosmetics.10-22-2009
20130219532PEPTIDES WITH ANTIFUNGAL ACTIVITIES - The present invention relates to antifungal and/or antibacterial peptides, especially antifungal peptides obtained from insect species, particularly lepidopterans. The present invention also provides methods of using these antifungal peptides to treat or prevent fungal growth for a variety of purposes, such as protecting plants from fungal infections; treating fungal infections of animals, especially humans; and prevention of food spoilage.08-22-2013
20090025097SHRNA AND SIRNA AND MIRNA EXPRESSION IN A LIVING ORGANISM UNDER CONTROL OF A CODON-OPTIMIZED REPRESSOR GENE - The present invention relates to a biological entity, notably a rat, carrying a regulator construct comprising a specific repressor gene and a responder construct comprising at least one segment corresponding to a short hairpin RNA (shRNA) or corresponding to complementary short interfering RNA (siRNA) strands or corresponding to miRNA, said at least one segment being under control of a promoter which contains an operator sequence corresponding to the repressor. The invention further relates to a method for preparing said biological entity and its use.01-22-2009
20110145941ANTIBODIES AND DERIVATIVES THEREOF - An anti-TrkA antibody is provided that comprises:06-16-2011
20080263689Methods for Identifying Modulators of Protein Kinase C-Epsilon (Pkce) and Method of Treatment of Aberrant Glucose Metabolism Associated Therewith - The present invention provides novel cell-based and animal-based assays for determining antagonists of PKCε and uses of the isolated antagonist compounds for modulating insulin clearance and secretion. The invention also provides novel animals and cells such as animals and cells suitable for use in the assays.10-23-2008
20090205061Taste Receptors Of The T1R Family From Domestic Cat - The present invention relates to the discovery of several genes of the domestic cat (08-13-2009
20090255003Diagnostic and therapeutic target SLC39A11 proteins for neurodegenerative diseases - The present invention discloses a dysregulation of the SLC39A11 gene and the protein products thereof in Alzheimer's disease patients. Based on this finding, the invention provides methods for diagnosing and prognosticating Alzheimer's disease in a subject, and for determining whether a subject is at increased risk of developing Alzheimer's disease. Furthermore, this invention provides therapeutic and prophylactic methods for treating and preventing Alzheimer's disease and related neurodegenerative disorders using the SLC39A11 gene and its corresponding gene products. Screening methods for modulating agents of neurodegenerative diseases are also disclosed.10-08-2009
20100263066INERT DNA SEQUENCES FOR EFFICIENT VIRAL PACKAGING AND METHODS OF USE - The instant invention provides an inert DNA sequence having a length of between about 0.5 kb and about 5 kb, wherein said isolated inert DNA sequence does not contain an open reading frame and which is suitable for efficient packaging of expression cassettes comprising a nucleic sequence encoding a therapeutic agent into viral vectors, as well as methods of selecting such inert DNA sequences. The invention also provides DNA constructs and medical composition comprising such inert DNA sequences, and kits and medical systems for delivering such DNA constructs and/or compositions.10-14-2010
20100192240REGULATION OF EXPRESSION OF PI3KBETA PROTEIN IN TUMORS - The present invention concerns the use of PI3Kβ protein and/or encoding gene for the screening for substances useful in the treatment of cancers, preferably breast cancers. The present invention also concerns a method for the diagnosis of malignant cell growth comprising the measuring the expression of PI3Kβ gene. The invention concerns also non-human transgenic animals as model study for human pathologies, preferably breast cancer, being transgenic for having altered PI3Kβ and Neu-T expression.07-29-2010
20100218264Genome editing in rats using zinc-finger nucleases - Disclosed herein are methods and compositions for genome editing of one or more loci in a rat, using fusion proteins comprising a zinc-finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding said fusion proteins are also provided, as are cells comprising said polynucleotides and fusion proteins.08-26-2010
20100212035Compositions and Methods for Producing Transgenic Mammals Having Recombinant Immunoglobulin Loci - The invention provides methods for the production of transgenic animals comprising a recombinant Ig locus, as well as transgenic antibodies derived therefrom. The methods involve meganuclease cleavage-stimulated homologous recombination in mammalian embryos.08-19-2010
20100169994ANIMAL MODEL - The invention relates to the use of a NK1−/− animal as a model for attention deficit hyperactivity disorder and related conditions, to markers for those conditions and to methods of treating such conditions.07-01-2010
20100229253CHARACTERIZATION OF THE I-SPOMI ENDONUCLEASE FROM FISSION YEAST - Isolated DNAs encoding the enzyme I-Spoml and its recognition and cutting site are provided. The DNA sequences can be incorporated in cloning and expression vectors, transformed cell lines and transgenic animals. The vectors are useful in gene mapping and site-directed insertion of genes.09-09-2010
20100235935PLASMA ANTI-DIABETIC NUCB2 PEPTIDE (PLADIN) AND USES THEREOF - The present invention provides pladin (plasma anti-diabetic nucb2 peptide) polypeptide and functional equivalent thereof that are useful for treating diabetes. The present invention provides a method of treating diabetes by administering to a subject nesfatin-1, pladin, or a functional equivalent thereof. The present invention also provides a method of treating diabetes by administering to subject plasmin inhibitors.09-16-2010
20090138978Use of bridge-1 and activators and inhibitors thereof in the treatment of insulin deficiency and diabetes - The present invention is directed to the use of Bridge-1 polynucleotides and Bridge-1 polypeptides, as well as activators and inhibitors of Bridge-1 activity, in the treatment of Bridge-1 mediated disorders, including diabetes.05-28-2009
20100223685IL-21 RECEPTOR KNOCKOUT ANIMAL AND METHODS OF USE THEREOF - A transgenic non-human mammal with a disruption in its IL-21 receptor gene is provided, along with methods of using the transgenic non-human mammal.09-02-2010
20100223684HUMANIN RECEPTOR OR HUMANIN-LIKE POLYPEPTIDE RECEPTOR - One aspect of the present invention is directed to search receptors based on the information of HN signaling pathways in order to find Humanin receptor or Humanin-like polypeptide receptor (HNR), and to reveal a mechanism of promoting or suppressing the intracellular signal transduction for neuroprotecting activity of HN and identify a compound involved in the mechanism. The aspect of the invention is directed to a method for screening of HNR agonist and HNR antagonist, to utilize the screened compound in development of a drug for the treatment of neurodegenerative disease, and to provide an assay system of AD neuronal cell death, and to provide methods for the compulsory expression of HNR gene or knocking-out of intracellular genes.09-02-2010
20110113498SIRT1 POLYMORPHIC VARIANTS AND METHODS OF USE THEREOF - Provided herein are Sirt1 polymorphic variants having a substitution at amino acid residue 107 or nucleotide 373. In certain embodiments, the Sirt1 polypeptide variants have a L107P substitution and the nucleic acid variants have a T373C substitution. Genetic and/or biochemical testing may be performed to identify whether a patient carries one of the disclosed polymorphic variants. Based on the polymorphic variant the patient carries, a medical practitioner may administer an appropriate therapy, such as a sirtuin activator.05-12-2011
20110093965PHOSPHOLIPASES, NUCLEIC ACIDS ENCODING THEM AND METHODS FOR MAKING AND USING THEM - The invention provides novel polypeptides having phospholipase activity, including, e.g., phospholipase A, B, C and D activity, patatin activity, phosphatidic acid phosphatases (PAP) and/or lipid acyl hydrolase (LAH) activity, nucleic acids encoding them and antibodies that bind to them. Industrial methods, e.g., oil degumming, and products comprising use of these phospholipases are also provided.04-21-2011
20110088106COMPOSITIONS AND METHODS FOR MEDIATING CELL CYCLE PROGRESSION - Hypercellular nonhuman organisms have functionally inactivated expression of a cyclin inhibitor gene, especially p27. The growth rate of nonhuman organisms are increased such that a desired size is attained more quickly than as compared to nonvariant organisms. Inhibitors of the p27 cyclin dependent kinase inhibitor protein or sequences encoding the protein modulate vertebrate cell cycle progression and increase the proportion of dividing cells to non-dividing cells in a population of treated cells. As the proportion of dividing cells increases, the cell population, e.g., hematopoietic progenitor (stem) cells, is more efficiently used for gene therapy applications. Transgenic animals and plants, and knockout alleles are provided.04-14-2011
20110072527I-CREI MEGANUCLEASE VARIANTS WITH MODIFIED SPECIFICITY, METHOD OF PREPARATION AND USES THEREOF - Method of preparing I-CreI meganuclease variants having a modified cleavage specificity, variants obtainable by said method and their applications either for cleaving new DNA target or for genetic engineering and genome engineering for non-therapeutic purposes. Nucleic acids encoding said variants, expression cassettes comprising said nucleic acids, vectors comprising said expression cassettes, cells or organisms, plants or animals except humans, transformed by said vectors.03-24-2011
20110035819CODON OPTIMIZED CFTR - A synthetic hCFTR DNA sequence has been developed that produces remarkably high levels of hCFTR mRNA and protein in dosed murine lungs and human cells in culture compared to the natural hCFTR cDNA. This synthetic DNA addresses problems inherent in some natural cDNAs, such as premature transcriptional truncation sites introduced during cDNA synthesis. Introns are initially present in mRNA until the mRNA is processed. cDNA made from processed mRNA is devoid of introns. Thus DNA sequences (exon junctions) are present in a cDNA molecule which are not present in cells in nature. These exon junctions may affect transcription. Methods for improving expression of CFTR are based on sequence changes in cDNA molecules. The improvement methods may be applied to other cDNA molecules which are refractory to in vivo expression efforts. Compositions embodying the sequence changes increase the production of both transgenic mRNA and protein from cDNA molecules.02-10-2011
20110030076Methods and compositions for targeted cleavage and recombination - Disclosed herein are methods and compositions for targeted cleavage of a genomic sequence, targeted alteration of a genomic sequence, and targeted recombination between a genomic region and an exogenous polynucleotide homologous to the genomic region. The compositions include fusion proteins comprising a cleavage domain (or cleavage half-domain) and an engineered zinc finger domain and polynucleotides encoding same. Methods for targeted cleavage include introduction of such fusion proteins, or polynucleotides encoding same, into a cell. Methods for targeted recombination additionally include introduction of an exogenous polynucleotide homologous to a genomic region into cells comprising the disclosed fusion proteins.02-03-2011
20110154520Vitamin K Epoxide Recycling Polypeptide VKORC1, a Therapeutic Target of Coumarin and Their Derivatives - The invention relates to a novel polypeptide vitamin K epoxide recycling polypeptide (VKORC1) as a target for coumarin and its derivatives. The invention further provides methods for identifying coumarin derivatives, and also claims VKORC1 polypeptides and VKORC1 nucleic acids containing a sequence abnormality associated with a VKORC1 associated deficiency such as warfarin resistance, wherein the VKORC1 polypeptides and VKORC1 nucleic acids can be used for diagnosing these deficiencies. Moreover, the invention relates to methods for identifying coumarin derivatives usable in pest control of rodents.06-23-2011
20110162096Gene Therapy Using Transposon-Based Vectors - Methods and compositions are presented for the administration of transposon-based vectors to an animal or human to provide gene therapy to the animal or human06-30-2011
20110258715ORGAN REGENERATION METHOD UTILIZING iPS CELL AND BLASTOCYST COMPLEMENTATION - It is revealed that an organ such as pancreas can be regenerated by utilizing a fact that the deficiency of an organ is complemented by injecting an induced pluripotent stem cell (iPS cell) into a developed blastocyst in a blastocyst complementation method. Thus, the present invention has solved the above-described object. This provides a method for producing a target organ, using an iPS cell, in a living body of a non-human mammal having an abnormality associated with a lack of development of the target organ in a development stage, the target organ produced being derived from a different individual mammal that is an individual different from the non-human mammal.10-20-2011
20110016544Methods and compositions for detecting and treating retinal diseases based on metargidin (ADAM-15) - The invention discloses multiple genes related to age-related macular degeneration (AMD) and/or phagocytosis by RPE cells of the eye, and methods and compositions for detecting and treating AMD and other retinal degenerative conditions based on these phagocytosis-related and/or AMD-related genes. Also provided are animal models useful for testing therapeutic compounds and treatment protocols for AMD, and gene arrays including polymorphic variants of phagocytosis-related and/or AMD-related genes, useful for genetic screening of nucleic acid samples from subjects to obtain profiles of polymorphic variant sequences in a plurality of genes associated with AMD.01-20-2011
20110010785PRODUCTS AND THEIR USE FOR THE DIAGNOSIS, PREVENTION AND/OR CARE OF HUMAN AND/OR ANIMAL PATHOLOGIES CHARACTERISED BY THE ANOMALOUS DEPOSITION OF B-AMYLOID AND/OR AMYLOID-LIKE SUBSTANCE IN HUMAN AND/OR ANIMAL ORGANS AND TISSUES, AND SCREENING METHOD FOR DETERMINING THE RISK OF SUCH PATHOLOGIES - The patent refers to a screening method carried out on biological material isolated from human and/or animal organisms for determining the risk of human and/or animal pathologies expressing an anomalous deposition of β-amyloid and/or amyloid-like substance in human and/or animal organs and tissues, based on the investigation of the punctiform mutation Ala>Val in position 2 of the β-protein (corresponding to the Ala673Val mutation precursor of the β-protein containing 770 amino acids) in homozygosis or in heterozygosis. The patent provides for the possibility of: (01-13-2011
20100242125METHODS FOR CORRECTING MITOTIC SPINDLE DEFECTS AND OPTIMIZING PREIMPLANTATION EMBRYONIC DEVELOPMENTAL RATES ASSOCIATED WITH SOMATIC CELL NUCLEAR TRANSFER IN ANIMALS - The present invention is directed to various methodologies to make NT a practical procedure for animals, specifically, primates including human and non-human primates. Furthermore, the methods and molecular components provided by the present invention provide a practical means for producing embryos with desired characteristics. In a specific embodiment, the methodology of the present invention comprises introducing nuclei having desired characteristics along with one or more molecular components into an enucleated egg, thus creating a nuclear transfer construct, culturing the egg to produce a viable embryo, transferring the embryo to the oviducts of a female, and producing a cloned animal.09-23-2010
20120151613MUTANTS OF ACTIVATION-INDUCED CYTIDINE DEAMINASE (AID) AND METHODS OF USE - The invention provides functional mutants of activation-induced cytidine deaminase (AID) protein that have increased activity as compared to a wild-type AID protein. The invention also provides nucleic acids encoding the functional AID mutants, and vectors and cells comprising the nucleic acids. The invention further provides methods of using the functional mutant AID proteins.06-14-2012
20100100979BIOMARKER SPECIFIC FOR CANCER - It is an object of the present invention to provide diagnostic reagents, pharmaceuticals and the like for particular diseases, and providing means that are useful in developing such reagents, pharmaceuticals and the like. The present invention provides a novel polypeptide and a specific partial peptide thereof, as well as a novel polynucleotide and a specific partial nucleotide thereof, that can be used as cancer-specific biomarkers; an expression vector for such a polynucleotide and a specific partial peptide thereof; a transformant incorporating such an expression vector; an antisense molecule, RNAi-inducing nucleic acid (e.g., siRNA), aptamer, or antibody for a cancer-specific biomarker, and a composition comprising the same; a mammalian cell or non-human mammal wherein the expression or a function of a cancer-specific biomarker is regulated; a measuring means (e.g., primer set, nucleic acid probe, antibody, aptamer) for a cancer-specific biomarker, and a reagent comprising them and the like.04-22-2010
20110265197Signatures and PCDeterminants Associated with Prostate Cancer and Methods of Use Thereof - The present invention provides methods of detecting cancer using biomarkers.10-27-2011
20090133136Inducible SIRNA expression cassette and method of use - An inducible siRNA expression polynucleotide and methods for its use are provided. The expression polynucleotide comprises a bicistronic expression cassette that encodes a repressor and a detectable marker, wherein the repressor controls expression of siRNA expression in the absence of an inducer.05-21-2009
20120117674METHOD FOR GENERATING REPLICATION DEFECTIVE VIRAL VECTORS THAT ARE HELPER FREE - Sequences are provided that are capable of directing circular adeno-associated virus replication, useful in vectors for providing therapeutic agents to a subject in need thereof. The vectors of the invention are particularly useful in the treatment of acute medical conditions requiring rapid gene expression. Further provided are methods for producing packaged defective viral vectors.05-10-2012
20120159660DESATURASES AND METHODS OF USING THEM FOR SYNTHESIS OF POLYUNSATURATED FATTY ACIDS - The amino acid and nucleic acid sequences of a Δ06-21-2012
20120073003APOBEC3 MEDIATED DNA EDITING - The present invention relates to methods and compositions for preventing the occurance or progression of a cancer or pre-cancerous condition associated with expression, or over-expression of human cytidine deaminases of the APOBEC3 family. The invention also relates to drug screening assays designed to identify compounds that regulate the activity, or level of expression, of hA3A, hA3C and hA3H. The invention further relates to transgenic mice, as well as cells derived from said mice, that have been genetically engineered to express, or over-express hA3A, hA3C and/or hA3H. Such mice may be utilized to screen for, or identify, compounds that modulate the activity, or expression, of the human cytidine deaminases. The present invention also provides topical compositions such as cosmetic lotion, crème, or sunscreen for use on the skin, which comprise one or more inhibitors of human cytidine deaminase activity. The present invention relates to a double stranded DNA obtained following opening up of its duplex structure, said DNA being edited with cellular protein normally or abnormally expressed in the nucleus of an eukaryotic cell. The mono stranded DNA derived from the said double stranded DNA is a part of the present invention.03-22-2012
20120124685COMPOSITIONS AND METHODS FOR GENE EXPRESSION AND CHROMATIN PROFILING OF INDIVIDUAL CELL TYPES WITHIN A TISSUE - The present invention provides compositions, methods, and kits for generating and isolating tagged nuclei of specific cell types with a high yield and purity. The compositions, methods, and kits provided herein comprise expressing in a cell a nuclear envelope tagging fusion polypeptide comprising a nuclear envelope targeting domain and an affinity reagent binding region. In some embodiments, expression of the fusion polypeptide is under the control of a cell type-specific promoter. Some embodiments also comprise expressing in a cell a biotin ligase, wherein the affinity reagent binding region comprises a biotin ligase accepting site, and wherein at least one of the nuclear envelope tagging fusion polypeptide and the biotin ligase is expressed under the control of a cell type-specific promoter.05-17-2012
20120180150INOSITOL 1,4,5-TRIPHOSPHATE RECEPTOR-BINDING PROTEIN AND ITS NON-HUMAN TRANSGENIC MAMMALIAN ANIMALS - The instant invention provides an inositol 1,4,5-triphaosphate receptor (IP3R)-binding protein and its transgenic non-human mammalian animal.07-12-2012
20120084872METHODS 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.04-05-2012
20120084871NPC1L1 ORTHOLOGUES - The present invention provides, in part, NPC1L1 from various species. Methods of using the NPC1L1 polypeptides and polynucleotide set forth herein, e.g., in screening assays, are also set forth.04-05-2012
20120240247JNK3 MODULATORS AND METHODS OF USE - The c-Jun NH09-20-2012
20110030077METHODS OF MODULATING THE ORGANIC SOLUTE AND STEROID TRANSPORTER (OSTalpha-OSTbeta) ACTIVITY AND TREATING ASSOCIATED CONDITIONS - The present invention is directed to methods of modulating bile acid and lipid transport via the organic solute and steroid transporter Ostα-Ostβ. The present invention is further directed to methods of treating a patient having dyslipidemia or a condition associated with altered bile acid homeostasis. Therapeutic agents and pharmaceutical compositions that modulate Ostα-Ostβ heteromeric complex formation and/or transport activity are also disclosed.02-03-2011
20120272349METHOD FOR CONSTRUCTING CHIMERIC RAT USING RAT EMBRYONIC STEM CELLS - The present invention provides a preparation method of a chimeric embryo and a chimeric rat, which is characterized by contacting a rat pluripotent stem cell and a host embryo in the presence of an ES cell differentiation inhibitor. The method includes (a) a step for contacting a fertilized host embryo collected from a female rat and a rat pluripotent stem cell in the presence of an ES cell differentiation suppressant, and (b) a step for culturing the host embryo in contact with the rat pluripotent stem cell to form a chimeric embryo.10-25-2012
20120331575TRANSGENIC NON-HUMAN ANIMALS - The invention provides a non-human transgenic animal comprising a transgene encoding angiogenin and food products comprising or obtained from the non-human transgenic animal and uses thereof.12-27-2012
20120090043TARGETS FOR RETROVIRUS ASSOCIATED DISEASES - The invention concerns an isolated complex comprising an HIV or HTLV protein and a human protein. Corresponding nucleic acids, vectors, host cells, host organisms, compositions, kits, medical uses, diagnostic uses, and methods of screening agents are also contemplated. Disclosed are 212 interactions between 19 retroviral proteins and 131 human proteins.04-12-2012
20100199366Gene therapy using transposon-based vectors - Methods and compositions are presented for the administration of transposon based vectors to an animal or human to provide gene therapy to the animal or human.08-05-2010
20110247089Organisms homozygous for targeted modification - Disclosed herein are homozygously modified organisms and methods of making and using these organisms.10-06-2011
20130179999NOVEL STRUCTURALLY DESIGNED shRNAs - Provided is an improved design of shRNA based on structural mimics of miR-451 precursors. These miR-451 shRNA mimics are channeled through a novel small RNA biogenesis pathway, require AGO2 catalysis and are processed by Drosha but are independent of DICER processing. This miRNA pathway feeds active elements only into Ago2 because of its unique catalytic activity. These data demonstrate that this newly identified small RNA biogenesis pathway can be exploited in vivo to produce active molecules.07-11-2013
20120255046IN VIVO TRANSDUCTION WITH A CHIMERIC AAV CAPSID PROTEIN - Recombinant adeno-associated viral (AAV) capsid proteins are provided. Methods for generating a library of recombinant adeno-associated viral capsid proteins are also provided.10-04-2012
20120255045INDUCIBLE SMALL RNA EXPRESSION CONSTRUCTS FOR TARGETED GENE SILENCING - The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic, particularly mam10-04-2012
20130174289Na+/Mg2+ Exchanger - The invention relates to methods to identify new SLC41A1 functions at the cell, tissue, organ and organism level. In part, it is related to methods useful in (a) identifying molecules that bind SLC41A1 polypeptides, which (b) modulate SLC41A1 related Na07-04-2013
20100319078Stem Cells Modified to Facilitate Threonine Catabolism - A mammalian cell comprising a recombinant, functional L-threonine 3-dehydrogenase (EC; TDH) gene, methods of making, and methods of use.12-16-2010
20130185818METHOD TO TRIGGER RNA INTERFERENCE - A method to generate siRNAs in vivo is described, as are constructs and compositions useful in the method. The method does not depend on the use of DNA or synthetic constructs that contain inverted duplications or dual promoters so as to form perfect or largely double-stranded RNA. Rather, the method depends on constructs that yield single-stranded RNA transcripts, and exploits endogenous or in vivo-produced miRNAs or siRNAs to initiate production of siRNAs. The miRNAs or siRNAs guide cleavage of the transcript and set the register for production of siRNAs (usually 21 nucleotides in length) encoded adjacent to the initiation cleavage site within the construct. The method results in specific formation of siRNAs of predictable size and register (phase) relative to the initiation cleavage site. The method can be used to produce specific siRNAs in vivo for inactivation or suppression of one or more target genes or other entities, such as pathogens.07-18-2013
20120030782Compositions and Methods for Generating Transgenic Animals - The present invention provides methods of altering gene expression of embryos to provide compositions and methods for efficient generation of transgenic animals. In particular, the present invention provides compositions and methods for generating germline transgenic animals by direct injection of nucleic acid molecules into animals.02-02-2012
20130198878ORGANISMS HOMOZYGOUS FOR TARGETED MODIFICATION - Disclosed herein are homozygously modified organisms and methods of making and using these organisms.08-01-2013
20090106852Influenza 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.04-23-2009

Patent applications in class Mammal

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