isolated antisense oligonucleotide, pharmaceutical composition, methods for inducing exon skipping of a gamma sarcoglycan rna, to relieve muscular dystrophy of type 2c belts, to inhibit the progression of lgmd2c-associated dystrophic pathology and to improve muscle function, and, kit.

摘要

The invention relates to one or more antisense polynucleotides and their use in pharmaceutical compositions in a strategy to induce exon skipping in the sarcoglycan gene in patients suffering from 2c-waist muscular dystrophy (lgm-d2c) or in patients with risk of such disease. The invention also provides methods for preventing or treating muscular dystrophy, for example lgmd2c, by exon skipping the gamma-sarcoglycan gene with the use of antisense polynucleotides. therefore, in some aspects, the invention provides an isolated antisense oligonucleotide, wherein the oligonucleotide specifically hybridizes to an exon-target region of a β-sarcoglycan rna. In another aspect, the invention provides a method for inducing exon skipping of a gamma-sarcoglycan RNA, which comprises delivering an antisense oligonucleotide or composition to a cell.