Antisense oligonucleotides for inducing exon skipping and methods of use thereof

摘要

An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.

主权项

1. An antisense oligonucleotide of 24 bases comprising SEQ ID NO:1, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′-O-methyl phosphorothioate oligoribonucleotide; or a pharmaceutically acceptable salt thereof.