RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy
The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.
1. A 2′-O-methyl phosphorothioate oligonucleotide of 25 bases in length, having the base sequence GUUGX 1 X 1 UX 1 X 1 GGUUX 1 UGAAGGUGUUX 1 (SEQ ID NO: 92), wherein each X 1 is 5-methyl-cytosine, or a pharmaceutically acceptable salt thereof.
2. A pharmaceutical composition, comprising the oligonucleotide of claim 1 and a pharmaceutically acceptable carrier.
3. A method for treating a patient with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) who has a mutation of the DMD gene that is amenable to exon 53 skipping, comprising administering to the patient the oligonucleotide of claim 1 , wherein the anti sense oligonucleotide induces exon 53 skipping.