Abstract
Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.
Full Text
What is claimed is:
Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.
Timeline
Filed
05/01/2026Published
08/20/2026Granted
Not AvailableIPC Codes(3)
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 47/60:the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
C12N 15/11:DNA or RNA fragments; Modified forms thereof (DNA or RNA not used in recombinant technology C07H 21/00)