Patent ID: 9415119
Filing Date: 2016-08-16
CPC Classification: A61K,A61P,C07K,C12N

Claim Text:
1. 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.