Abstract: The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.

Abstract: Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.

Abstract: The present disclosure relates to compositions, methods and processes for the formulation and for the administration of a gene therapy agent using parvovirus e.g., adeno-associated virus (AAV) to the CNS, CNS tissues, CNS structures or CNS cells.

Abstract: The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

Abstract: The disclosure relates to nucleic acid expression cassettes and vectors for the treatment of neurodegenerative disorders. Methods of treating neurodegenerative disorders such as Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, cognitive impairment, and mild cognitive impairment are also provided.

Abstract: The disclosure relates to nucleic acid expression cassettes for the treatment of neurodegenerative disorders. Methods of treating neurodegenerative disorders such as Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, cognitive impairment, and mild cognitive impairment are also provided.

Abstract: Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.

Abstract: The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

Abstract: The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.

Abstract: The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.

Abstract: The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

Abstract: A steering system for a vehicle includes a steering column, a steering wheel rotatable about an axis between rotational positions, and a magnetic paddle shift system for selecting a gear of a transmission. A sensor assembly has a sensor array. First and second paddle shifters are rotatable with the steering wheel and extend from a proximal end to a distal end. First and second magnets are disposed at the proximal end of the first and second paddle shifters, respectively, in alignment with the sensor array in any of the rotational positions. Actuation of the first and second paddle shifters move, respectively, the first and second magnets relative to the sensor array between first and second positions, the magnets being closer to the sensor array in the second position. The sensor array is configured to sense the first and second magnets in the second position for any of the rotational positions.

Abstract: A steering system for a vehicle includes a steering column, a steering wheel rotatable about an axis between rotational positions, and a magnetic paddle shift system for selecting a gear of a transmission. A sensor assembly has a sensor array. First and second paddle shifters are rotatable with the steering wheel and extend from a proximal end to a distal end. First and second magnets are disposed at the proximal end of the first and second paddle shifters, respectively, in alignment with the sensor array in any of the rotational positions. Actuation of the first and second paddle shifters move, respectively, the first and second magnets relative to the sensor array between first and second positions, the magnets being closer to the sensor array in the second position. The sensor array is configured to sense the first and second magnets in the second position for any of the rotational positions.

Abstract: Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.

Abstract: The present disclosure relates to compositions, methods and processes for the formulation and for the administration of a gene therapy agent using parvovirus e.g., adeno-associated virus (AAV) to the CNS, CNS tissues, CNS structures or CNS cells.

Abstract: The invention relates to compositions and methods for the preparation, administration, manufacture and therapeutic use of viral particles for the treatment of CNS disorders.

Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding payloads for the treatment of CNS disorders.

Abstract: The invention is based, in part, on the discovery that secretin and secretin analogs can be used to treat affective disorders that include symptoms of anxiety. Secretin and secretin analogs can be administered to patients to treat affective disorders, such as mood, psychotic, mental, substance-abuse withdrawal, eating, and sexual disorders, as well as other disorders with attendant sensory processing dysfunction, including symptoms of anxiety and/or fear. In addition, secretin and secretin analogs can be administered to improve cognition, including learning and memory, as well as to lessen the effects of conditioned startle. Secretin has the added benefit of causing no negative side effects upon administration and being easy to administer.