OLIGONUCLEOTIDE COMPRISING AN INOSINE FOR TREATING DMD

摘要

Provided is an oligonucleotide comprising an inosine and/or a nucleotide containing a base able to form a wobble base pair, wherein the oligonucleotide comprises a sequence which is complementary to at least part of a dystrophin pre-mRNA exon or at least part of a non-exon region of a dystrophin pre-mRNA said part being a contiguous stretch comprising at least 8 nucleotides. The oligos can be used to treat Duchenne Muscular Dystrophy.