Abstract: The invention relates to a novel Glutamic Acid Decarboxylase (GAD). More specifically, novel DNA and protein sequences relating to GAD. Additionally, the invention discloses a novel composition and related methods for treating neurodegenerative diseases such as Parkinson's disease, Alzheimer's disease, epilepsy, and the like, using viral and non-viral delivery systems that deliver therapeutic agents to specific regions of the brain.

Abstract: The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with a neurodegenerative diseases that is characterized by an excess buildup of buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.

Abstract: The invention relates to a novel Glutamic Acid Decarboxylase (GAD). More specifically, novel DNA and protein sequences relating to GAD. Additionally, the invention discloses a novel composition and related methods for treating neurodegenerative diseases such as Parkinson's disease, Alzheimer's disease, epilepsy, and the like, using viral and non-viral delivery systems that deliver therapeutic agents to specific regions of the brain. More specifically, using an adeno-associated viral vector to deliver a nucleotide sequence encoding a novel glutamic acid decarboxylase (GAD) to specific regions of the brain that are over stimulated or disinhibited in various diseases, including neurodegenerative diseases.

Abstract: The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with a neurodegenerative diseases that is characterized by an excess buildup of buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.

Abstract: The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the hippocampus, subthalamic nucleus of the basal ganglia, mesaphilia and thalamus.

Abstract: The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the hippocampus, subthalamic nucleus of the basal ganglia, mesaphilia and thalamus.

Abstract: The present invention relates to a method for delivering a nucleic acid sequence encoding neuropeptide Y, or a derivative or functional fragment thereof, to a mammalian nervous system target cell. The expression of exogenous NPY, or a derivative or a functional fragment thereof in the target cell(s) provides therapeutic benefit for subjects afflicted with a neurological disorder.

Abstract: The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the hippocampus, subthalamic nucleus of the basal ganglia, mesaphilia and thalamus.

Abstract: The present invention relates to a method for delivering a nucleic acid sequence encoding neuropeptide Y, or a derivative or functional fragment thereof, to a mammalian nervous system target cell. The expression of exogenous NPY, or a derivative or a functional fragment thereof in the target cell(s) provides therapeutic benefit for subjects afflicted with a neurological disorder.

Abstract: The present invention related to methods and compositions comprising recombinant vectors comprising chimeric capsids and recombinant pseudotyped vectors with non-native capsid protein(s). The recombinant vectors of the invention confer an altered tropism that permits selective targeting of desired cells.

Abstract: The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the hippocampus, subthalamic nucleus of the basal ganglia, mesaphilia and thalamus.

Abstract: The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the subthalmic nucleus of the basal ganglia, mesaphilia and thalamus.

Abstract: The invention relates to a novel Glutamic Acid Decarboxylase (GAD). More specifically, novel DNA and protein sequences relating to GAD. Additionally, the invention discloses a novel composition and related methods for treating neurodegenerative diseases such as Parkinson's disease, Alzheimer's disease, epilepsy, and the like, using viral and non-viral delivery systems that deliver therapeutic agents to specific regions of the brain.

Abstract: The present application relates to novel methods and compositions for treating metabolic disorders. Some aspects pertain to the use of gene therapy to treat diseases related to metabolic disorders, such as diabetes, obesity, high blood pressure, wasting syndrome, cachexia and atherogenic dyslipidemia. The present application also pertains to the use of vectors such as a recombinant adeno-associated virus (AAV) to deliver a at least a portion of a gene that can increase or decrease expression of a therapeutic protein of interest, e.g., in cells in a specific region of the brain associated with metabolic disorder. The present application also discloses the use of vectors such as a recombinant adeno-associated virus for the delivery of small interference RNA's (siRNAs) capable of decreasing expression of a deleterious protein involved in the disorder. Other related aspects, including compositions related to such methods, are also disclosed.

Abstract: The invention relates to a novel Glutamic Acid Decarboxylase (GAD). More specifically, novel DNA and protein sequences relating to GAD. Additionally, the invention discloses a novel composition and related methods for treating neurodegenerative diseases such as Parkinson's disease, Alzheimer's disease, epilepsy, and the like, using viral and non-viral delivery systems that deliver therapeutic agents to specific regions of the brain. More specifically, using an adeno-associated viral vector to deliver a nucleotide sequence encoding a novel glutamic acid decarboxylase (GAD) to specific regions of the brain that are over stimulated or disinhibited in various diseases, including neurodegenerative diseases.

Abstract: The present application relates to novel methods for treating obesity. Some aspects pertain to the use of gene therapy to treat diseases related to metabolic dysfunction, such as diabetes, obesity, high blood pressure, and atherogenic dyslipidemia. The present application also pertains to the use of vectors such as a recombinant adeno-associated virus (AAV) to deliver a gene that can increase or decrease expression of a therapeutic protein of interest, e.g., in cells in a specific region of the brain associated with metabolic dysfunction. The present application also discloses the use of vectors such as a recombinant adeno-associated virus for the delivery of small interference RNA's (siRNAs) capable of decreasing expression of a deleterious protein involved in the disorder. Other related aspects, including compositions related to such methods, are also disclosed.

Abstract: The invention relates to novel Glutamic Acid Decarboxylases (GAD). More specifically, novel DNA and protein sequences relating to GAD. Additionally, the invention discloses a novel composition and related methods for treating neurodegenerative diseases such as Parkinson's disease, Alzheimer's disease, epilepsy, and the like, using viral and non-viral delivery systems that deliver therapeutic agents to specific regions of the brain. More specifically, using an adeno-associated viral vector to deliver a nucleotide sequence encoding a novel glutamic acid decarboxylase (GAD) to specific regions of the brain that are over stimulated or disinhibited in various diseases, including neurodegenerative diseases.

Abstract: The invention provides methods and compositions of an upstream regulatory element (PINK-1 promoter) operably linked to an expressible gene, wherein the expression of the expressible gene is driven by the upstream regulatory element.

Abstract: The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with a neurodegenerative diseases that is characterized by an excess buildup of buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.

Abstract: The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the subthalmic nucleus of the basal ganglia, mesaphilia and thalmus.