Abstract: The present invention is directed to compositions methods and kits for regulation of gene therapies, including, without limitation, reversible gene therapies and allele-specific therapies.

Abstract: In one example, a method includes selecting, by one or more processors and based on a representation of sensed electrical signals for a particular patient and a plurality of representations of sensed electrical signals for a plurality of other patients, a combination of electrodes of a plurality of combinations of one or more implantable electrodes for delivery of electrical stimulation to the particular patient.

Abstract: In some examples, a processor of a system evaluates a therapy program based on a score determined based on a volume of tissue expected to be activated (“VTA”) by therapy delivery according to the therapy program. The score may be determined using an efficacy map comprising a plurality of voxels that are each assigned a value. In some examples, the efficacy map is selected from a plurality of stored efficacy maps based on a patient condition, one or more patient symptoms, or both the patient condition and one or more patient symptoms. In addition, in some examples, voxels of the efficacy map are assigned respective values that are associated with a clinical rating scale.

Abstract: 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.

Abstract: A therapeutic window for at least one electrode of a medical system may be determined based on a volume of tissue expected to be activated (“VTA”) by electrical stimulation delivered by the at least one electrode. In some examples, a processor determines the therapeutic window for a particular electrode by at least determining an efficacy threshold based on the VTA expected to result from the delivery of electrical stimulation according to a set of electrical stimulation parameter values including the stimulation parameter at the efficacy threshold, and determining an adverse-effects threshold based on the VTA expected to result from the delivery of electrical stimulation according to a set of electrical stimulation parameter values including the stimulation parameter at the adverse-effects threshold.

Abstract: 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.

Abstract: 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.

Abstract: 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.

Abstract: 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.

Abstract: Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatto and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.

Abstract: Methods for delivering first and second fluid compositions to a target location of a subject include delivering the first composition from reservoir of an implantable infusion pump and delivering the second composition through a catheter access port of the implantable infusion device. The access port and reservoir are fluidly coupled to a catheter having a delivery region implanted in the target region. The methods more fully realize the therapeutic potential of infusion devices having both an access port and a reservoir. Strategic use of different agents that compliment the function of each other delivered via either the access port or the reservoir can result in enhanced therapeutic potential.

Abstract: 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.

Abstract: Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatto and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.

Abstract: 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.

Abstract: 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.

Abstract: Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatto and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.

Abstract: 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.

Abstract: 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.

Abstract: 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.

Abstract: 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.