Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping |
| |
Authors: | Goyenvalle Aurélie Vulin Adeline Fougerousse Françoise Leturcq France Kaplan Jean-Claude Garcia Luis Danos Olivier |
| |
Institution: | Généthon & CNRS UMR 8115, 1, rue de l'Internationale, Evry, 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. |
| |
Keywords: | |
本文献已被 PubMed 等数据库收录! |
|