Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Abstract

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.

1 . (canceled) 
     
     
         2 . An antisense oligonucleotide of 30 bases comprising the base sequence CUCCAACAUC AAGGAAGAUG GCAUUUCUAG (SEQ ID NO: 181), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide. 
     
     
         3 . The antisense oligonucleotide of  claim 2 , wherein the antisense oligonucleotide is chemically linked to one or more moieties or conjugates that enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotide. 
     
     
         4 . The antisense oligonucleotide of  claim 2 , wherein the oligonucleotide is chemically linked to a polyethylene glycol chain. 
     
     
         5 . A pharmaceutical composition comprising an antisense oligonucleotide of 30 bases comprising the base sequence CUCCAACAUC AAGGAAGAUG GCAUUUCUAG (SEQ ID NO: 181), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and a pharmaceutically acceptable carrier. 
     
     
         6 . The pharmaceutical composition of  claim 5 , wherein the antisense oligonucleotide is chemically linked to one or more moieties or conjugates that enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotide. 
     
     
         7 . The pharmaceutical composition of  claim 5 , wherein the oligonucleotide is chemically linked to a polyethylene glycol chain.