Sarepta Therapeutics, Inc.
COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

Abstract:

Methods for treating Duchenne muscular dystrophy using conjugates of cell penetrating peptides to antisense oligonucleotide that induce exon skipping in the human dystrophin gene. In one embodiment, the cell penetrating peptide is an arginine-rich peptide comprising six contiguous arginine residues and the antisense oligonucleotide is eteplirsen, golodirsen or casimersen.