Multiple exon skipping compositions for dmd

Details Multiple exon skipping compositions for dmd Multiple exon skipping compositions for dmd

C - Chemistry, Metallurgy

12

N

C12N 15/113 (2010.01)

Patent

CA 2740328

Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to in-duce exon skipping, and methods of use thereof to treat muscular dystrophy.

La présente invention concerne des molécules antisens capables de se lier à un site cible sélectionné dans le gène de la dystrophine humaine pour induire un saut dexon, et concerne également des procédés dutilisation associés pour traiter la dystrophie musculaire.

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