Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Abstract

Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.

1 . (canceled) 
     
     
         2 . An isolated antisense oligonucleotide 20 to 50 nucleotides comprising at least 17 bases of the sequence CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO:192), or an equivalent oligonucleotide, wherein said oligonucleotide, or its equivalent, induces exon 53 skipping in the human dystrophin pre-mRNA, and wherein the oligonucleotide comprises a modification, said modification comprising a base substitution of U by T. 
     
     
         3 . An isolated antisense oligonucleotide of 24-50 nucleotides in length, said oligonucleotide comprising a sequence which is complementary to a target nucleic acid sequence of the human exon 53 pre-mRNA, wherein said target nucleic acid sequence comprises a 24 nucleotide sequence that is complementary to the sequence CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO:192), and wherein the oligonucleotide comprises a modification, said modification comprising a base substitution of U by T.