Patent ID: 9434928
Filing Date: 2016-09-06
CPC Classification: A61K,A61M,A61P,C07K,C12N

Claim Text:
1. A method of improving muscle function in a patient afflicted with limb girdle muscular dystrophy type 2D (LGMD 2D) comprising the step of perfusing the vasculature of a limb of the patient with an infectious encapsidated recombinant adeno-associated virus (AAV) comprising a AAVrh.74 capsid polynucleotide of SEQ ID NO:2, wherein the genome of the recombinant AAV comprises a gene expression cassette comprising the polynucleotide of SEQ ID NO: 3 encoding an alpha-sarcoglycan under the transcriptional control of a promoter, said cassette flanked by one or more AAV inverted terminal repeats, and wherein the genome lacks AAV rep and cap DNA.