Patent ID: 11911440
Assignee: GENZYME CORPORATION
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A method of treating spinal muscular atrophy (SMA) comprising administering to a subject in need thereof a therapeutically effective amount of recombinant adeno-associated virus (rAAV) virions comprising a self-complementary adeno-associated virus (scAAV) vector comprising a heterologous nucleic acid construct, wherein the heterologous nucleic acid construct comprises:
a. a first AAV2 inverted terminal repeat (ITR);
b. a cytomegalovirus enhancer/chicken-β actin (CBA) promoter;
c. a polynucleotide encoding a survival motor neuron (SMN) protein comprising the amino acid sequence of SEQ ID NO: 2; and
d. a second AAV2 ITR, and
wherein the rAAV virions comprise an AAV8 or AAV9 capsid, and
wherein the scAAV virions are administered via intrathecal injection.