Patent ID: 9499817
Filing Date: 2016-11-22
CPC Classification: A61K,A61P,C12N

Claim Text:
1. A method of inducing exon-skipping of a gamma sarcoglycan RNA, comprising delivering to a human muscle cell an antisense polynucleotide, wherein the polynucleotide is about 5 to about 50 nucleotides in length and specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO: 1), exon 5 (SEQ ID NO: 2), exon 6 (SEQ ID NO: 3), exon 7 (SEQ ID NO: 4) and a combination thereof, thereby inducing exon-skipping of the gamma sarcoglycan RNA.