OLIGONUCLEOTIDE COMPRISING AN INOSINE FOR TREATING DMD

摘要

The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.

主权项

1. An isolated antisense oligonucleotide 13 to 50 nucleotides in length comprising an inosine base, wherein said oligonucleotide is capable of binding to an exon of the human dystrophin pre-mRNA so as to induce skipping of said exon.