METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA

Abstract

The. invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

1 - 15 . (canceled) 
     
     
         16 . An antisense oligonucleotide of 20 to 24 nucleotides in length, wherein the antisense oligonucleotide comprises at least 18 consecutive bases of a base sequence of the sequence CUGUUGCCUCCGGUUCUGAAGGUG (SEQ ID NO: 115), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino phosphorodiamidate antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 53 skipping of human dystrophin pre-mRNA. 
     
     
         17 . The antisense oligonucleotide of  claim 16  that is 21 nucleotides in length. 
     
     
         18 . A pharmaceutical composition, comprising the oligonucleotide of  claim 16  and a pharmaceutically acceptable excipient. 
     
     
         19 . The pharmaceutical composition, comprising the oligonucleotide of  claim 17  and a pharmaceutically acceptable excipient. 
     
     
         20 . A method for treating Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD), comprising administering to a subject a therapeutically effective amount of the oligonucleotide of  claim 16 . 
     
     
         21 . A method for treating Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD), comprising administering to a subject a therapeutically effective amount of the oligonucleotide of  claim 17 .