Oligonucleotide comprising an inosine for treating dmd

Details Oligonucleotide comprising an inosine for treating dmd Oligonucleotide comprising an inosine for treating dmd

C - Chemistry, Metallurgy

12

N

C12N 15/113 (2010.01) C12N 15/11 (2006.01)

Patent

CA 2759899

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.

La présente invention porte sur un oligonucléotide comprenant une inosine et/ou un nucléotide contenant une base capable de former une paire de bases Wobble ou un équivalent fonctionnel à celle-ci, dans laquelle l'oligonucléotide ou un équivalent fonctionnel de celui-ci comprend une séquence qui est complémentaire à au moins une partie de l'exon du pré-ARNm de la dystrophine ou à au moins une partie d'une région nonexon d'un pré-ARNm de la dystrophine, ladite partie étant un étirement adjacent contenant au moins 8 nucléotides. L'invention porte également sur l'utilisation dudit oligonucléotide pour prévenir ou traiter une dystrophie musculaire de Duchenne (DMD) ou une dystrophie musculaire de Becker (BMD).

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