Rescue of Dystrophic Muscle Through U7 snRNA-Mediated Exon Skipping

Author:

Goyenvalle Aurélie12,Vulin Adeline12,Fougerousse Françoise12,Leturcq France12,Kaplan Jean-Claude12,Garcia Luis12,Danos Olivier12

Affiliation:

1. Généthon & CNRS UMR 8115, 1, rue de l'Internationale, Evry, France.

2. Laboratoire de Biochimie et de Génétique Moléculaire, Hôpital et Institut Cochin, 123 boulevard de Port-Royal, Paris, France.

Abstract

Most mutations in the dystrophin gene create a frameshift or a stop in the mRNA and are associated with severe Duchenne muscular dystrophy. Exon skipping that naturally occurs at low frequency sometimes eliminates the mutation and leads to the production of a rescued protein. We have achieved persistent exon skipping that removes the mutated exon on the dystrophin messenger mRNA of the mdx mouse, by a single administration of an AAV vector expressing antisense sequences linked to a modified U7 small nuclear RNA. We report the sustained production of functional dystrophin at physiological levels in entire groups of muscles and the correction of the muscular dystrophy.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

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