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US 9,415,119 B2Granted
Gene therapy for neurodegenerative disorders
AssigneeGenzyme Corporation
InventorsMarco A. Passini, Lamya Shihabuddin, Seng H. Cheng
Life of this patent
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Abstract
Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
Claims7
A method of modulating motor function in a subject with spinal muscular atrophy (SMA) comprising administering a therapeutically effective amount of a pharmaceutical composition comprising a recombinant adeno-associated virus (rAAV) virion to the central nervous system of the subject;
- wherein the rAAV virion comprises a self-complementary adeno-associated virus (scAAV) vector comprising a polynucleotide encoding a survival motor neuron (SMN) protein, wherein the rAAV virion comprises an AAV9 capsid, wherein the composition is administered via administration into at least one region of the deep cerebellar nuclei of the cerebellum, via direct spinal cord injection, via intracerebroventricular injection, or via intrathecal injection.
Dependencies are inferred from the claim text.