Exon skipping compositions for treating muscular dystrophy

Abstract

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.

1 - 20 . (canceled) 
     
     
         21 . A morpholino antisense oligonucleotide of 20 to 25 bases comprising at least 20 consecutive nucleotides 100% complementary to a dystrophin exon 44 target region designated as annealing site H44A(+77+101), or pharmaceutically acceptable salt thereof. 
     
     
         22 . A morpholino antisense oligonucleotide of 20 to 25 bases comprising at least 20 consecutive nucleotides of SEQ ID NO: 9, or pharmaceutically acceptable salt thereof. 
     
     
         23 . The antisense oligonucleotide or pharmaceutically acceptable salt thereof of  claim 22 , 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. 
     
     
         24 . The antisense oligonucleotide or pharmaceutically acceptable salt thereof of  claim 22 , wherein the oligonucleotide is chemically linked to a polyethylene glycol moiety. 
     
     
         25 . A pharmaceutical composition comprising an antisense oligonucleotide of 25 bases comprising SEQ ID NO: 9, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and a pharmaceutically acceptable carrier, or pharmaceutically acceptable salt thereof. 
     
     
         26 . The pharmaceutical composition or pharmaceutically acceptable salt thereof of  claim 25 , 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. 
     
     
         27 . The pharmaceutical composition or pharmaceutically acceptable salt thereof of  claim 25 , wherein the oligonucleotide is chemically linked to a polyethylene glycol moiety.