abstract |
The invention relates to antisense oligonucleotides hybridizable to target regions in exons 53, 33, 45 or 50 of the Dystrophin gene, which oligonucleotides may comprise a non-natural backbone containing non-natural sugar moieties and/or non-natural inter-nucleotide linkages, and the use of such oligonucleotides in the treatment of muscular dystrophy, e.g. Duchenne Muscular Dystrophy. |