OLIGONUCLÉOTIDES ANTISENS QUI SE LIENT À L'EXON 51 DU PRÉ-ARNM DE LA DYSTROPHINE HUMAINE

摘要

The present invention relates to a therapeutic antisense oligonucleotide which binds to exon 51 of the human dystrophin pre-mRNA to induce exon skipping, and conjugates and compositions thereof for the treatment of DMD.