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.

We claim: 
     
         1 . 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. 
     
     
         2 . An antisense molecule according to  claim 1  capable of inducing exon skipping in exons 3, 4, 8, 10 to 16, 19 to 40, 42 to 44, 46, 47 and 50 to 53 of the dystrophin gene. 
     
     
         3 . A combination of two or more antisense molecules according to  claim 1  or  2  capable of binding to a selected target to induce exon skipping in the dystrophin gene. 
     
     
         4 . A combination or two or more antisense molecules according to  claim 3  selected from Table 1B. 
     
     
         5 . A combination of two or more antisense molecules according to  claim 1  or  2  joined together to form a “weasel”, wherein said weasel is capable of binding to a selected target to induce exon skipping in the dystrophin gene. 
     
     
         6 . A combination of two or more antisense molecules according to  claim 5  selected from Table 1C. 
     
     
         7 . The antisense molecule according to any one of  claims 1  to  6 , capable of binding to a selected target site, wherein the target site is an rnRNA splicing site selected from a splicer donor site, splice acceptor sites or exonic splicing enhancer elements. 
     
     
         8 . A method of treating muscular dystrophy in a patient comprising administering to the patient a composition comprising an antisense molecule according to anyone of  claims 1  to  6 . 
     
     
         9 . A pharmaceutical or therapeutic composition for the treatment of muscular dystrophy in a patient comprising (a) at least an antisense molecule according to any one of  claims 1  to  6 , and (b) one or more pharmaceutically acceptable carriers and/or diluents. 
     
     
         10 . The composition according to  claim 9 , comprising about 20 nM to 600 nM of the antisense molecule. 
     
     
         11 . The use of an antisense molecule according to any one of  claims 1  to  6  for the manufacture of a medicament for modulation of muscular dystrophy. 
     
     
         12 . An antisense molecule according to any one of  claims 1  to  6  for use in antisense molecule based therapy. 
     
     
         13 . An antisense molecule according to any one of  claims 1  to  6  as herein before described with reference to the examples. 
     
     
         14 . A kit comprising at least one antisense molecule according to any one of  claims 1  to  6 , a suitable carrier and instructions for its use.