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 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of GGG AUC CAG UAU ACU UAC AGG CUC C (SEQ ID NO: 175), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 50 skipping; or a pharmaceutically acceptable salt thereof.