Antisense-oligonukleotider til inducering af exon-skipping og fremgangsmåder til anvendelse deraf

摘要

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.