Patent application number | Description | Published |
20080280843 | Methods 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 |
20090042824 | Methods 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 |
20090060987 | DEVICES, SYSTEMS AND METHODS FOR IMPROVING MEMORY AND/OR COGNITIVE FUNCTION THROUGH BRAIN DELIVERY OF SIRNA - The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The present invention also provides valuable small interfering RNA vectors, and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject. | 03-05-2009 |
20100063134 | TREATMENT 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 |
20100113351 | Therapeuting Compositions Comprising an RNAi Agent and a Neurotrophic Factor and Methods of Use Thereof - The invention provides novel combination therapies for treating Huntington's disease which comprise a) BDNF or suitable fragments thereof and b) agents capable of causing inhibition of a gene responsible for the neurodegenerative disease. The invention provides nucleic acid sequences, methods, and systems suitable for applications of these combination therapies. | 05-06-2010 |
20100114206 | Patient Interface Device and Therapy Delivery System - A method and system for diagnosing a medical condition, alerting a patient that a therapy is impending, and allowing the patient to override or modify the impending therapy are provided. The system may include an implantable medical device (“IMD”) and a patient interface device. The IMD may sense a physiological parameter within a patient, determine whether the physiological parameter qualifies for therapy based on a therapy criteria, and alert the patient via the patient interface device that therapy is impending if the physiological parameter qualifies for therapy. The patient may be given an opportunity to respond to the alert with the patient interface device and send an therapy modification indication if the therapy is not needed or wanted. The IMD may wait a predetermined period of time for the patient response and deliver the therapy if the patient response is not transmitted before expiration of the predetermined period of time or withhold the therapy if the patient overrides the delivery of the therapy within the predetermined period of time. The IMD may adjust one or more operational settings applied during its operation in response to the activity of the system. The patient interface device may be configured to avoid accidental override or modification of the delivery of the therapy. | 05-06-2010 |
20100158869 | COMPOSITIONS, DEVICES AND METHODS FOR TREATMENT OF HUNTINGTON'S DISEASE THROUGH INTRACRANIAL DELIVERY OF SIRNA - The present invention provides devices, small interfering RNAs, 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, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient. | 06-24-2010 |
20100184835 | Devices, Systems and Methods for Improving Memory and/or Cognitive Function Through Brain Delivery of Sirna - The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The present invention also provides valuable small interfering RNA vectors, and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject. | 07-22-2010 |
20100284990 | Compositions and Methods for Making Therapies Delivered by Viral Vectors Reversible for Safety and Allele-Specificity - The present invention is directed to compositions, methods and kits for regulation of gene therapies for Huntington's Disease, including, without limitation, reversible gene therapies and allele-specific therapies. | 11-11-2010 |
20100325746 | Methods 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 |
20110052666 | Compositions, Methods, and Systems for SIRNA Delivery - The instant invention provides silencer expression cassettes for delivery of RNAi therapy. The silencer cassettes of the instant invention encode an RNAi agent and lack means for replication within a host cell, inverted terminal repeats and further lacking one or more selectable markers. Optionally, the silencer cassettes lack CpG dinucleotides and/or the RNAi agent is within a scaffold of a naturally occurring miRNA. The composition comprising the silencer cassettes of the instant invention, as well as methods of use and suitable systems are also provided. | 03-03-2011 |
20110160707 | COMPOSITIONS, DEVICES AND METHODS FOR TREATMENT OF HUNTINGTON'S DISEASE THROUGH INTRACRANIAL DELIVERY OF SIRNA - The present invention provides devices, small interfering RNAs, 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, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient. | 06-30-2011 |
20110213328 | Methods and Systems for Treatment of Neurological Diseases of the Central Nervous System - The present invention is directed to methods and systems for the treatment of inborn genetic errors or other defects that cause deficiencies of active enzymes or proteins within the cells of the central nervous system. Such methods and systems generally comprise an implantable catheter system designed for the chronic delivery of specially formulated proteins to intrathecal, intracerebroventricular, and/or intraparenchymal regions of the central nervous system. The invention has application in the neuropathic aspects of the broad category of lysosomal storage diseases. These genetic based diseases are the result of insufficient enzyme activity to catabolize specific substances, which thereby accumulate in the cellular lysosomes. | 09-01-2011 |
20120093916 | Devices, Systems and Methods for Improving Memory and/or Cognitive Function Through Brain Delivery of SIRNA - The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The present invention also provides valuable small interfering RNA vectors, and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject. | 04-19-2012 |
20120172409 | METHODS 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 |
20120253261 | SYSTEMS AND METHODS FOR OPTOGENETIC MODULATION OF CELLS WITHIN A PATIENT - Cells within a patient are optogenetically modulated to treat various neurological disorders. In one example, a method includes delivering a viral vector including a genetic agent encoding for one or more light-sensitive proteins to a delivery site within a patient. The viral vector includes retrograde and/or anterograde transport properties such that the viral vector is configured to transduce the genetic agent into cells at the delivery site and into cells in a plurality of sites proximal and remote to the delivery site. A bioelectrical signal(s) related to a neurological condition of the patient is sensed, e.g. using an implanted electrode. Optical stimulation is delivered to cells transduced with the genetic agent by the viral vector to treat the neurological condition of the patient. | 10-04-2012 |
20130287275 | Volume Of Efficacy Model Capture - A system and method for determining, planning, and/or administering a therapy of a selected material from a catheter to a selected specific region of an anatomy is disclosed. The catheter can be used to deliver a drug to the patient to achieve a volume of efficacy (VOE) within a selected target, region, or volume. The drug delivered can be used for treating at least a symptom in the patient. | 10-31-2013 |