Sarepta Therapeutics, Inc.
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPY

Abstract:

Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 50 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): ##STR00001## or a pharmaceutically acceptable salt thereof, wherein T, Nu, n, and R.sup.100 are defined herein.