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- Title
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
- 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.
- Publication
Science (New York, N.Y.), 2004, Vol 306, Issue 5702, p1796
- ISSN
1095-9203
- Publication type
Journal Article
- DOI
10.1126/science.1104297