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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.
Science. 2004 Dec 03; 306(5702):1796-9.Sci

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.

Authors+Show Affiliations

Généthon & CNRS UMR 8115, 1, rue de l'Internationale, Evry, France.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

15528407

Citation

Goyenvalle, Aurélie, et al. "Rescue of Dystrophic Muscle Through U7 snRNA-mediated Exon Skipping." Science (New York, N.Y.), vol. 306, no. 5702, 2004, pp. 1796-9.
Goyenvalle A, Vulin A, Fougerousse F, et al. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science. 2004;306(5702):1796-9.
Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J. C., Garcia, L., & Danos, O. (2004). Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science (New York, N.Y.), 306(5702), 1796-9.
Goyenvalle A, et al. Rescue of Dystrophic Muscle Through U7 snRNA-mediated Exon Skipping. Science. 2004 Dec 3;306(5702):1796-9. PubMed PMID: 15528407.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. AU - Goyenvalle,Aurélie, AU - Vulin,Adeline, AU - Fougerousse,Françoise, AU - Leturcq,France, AU - Kaplan,Jean-Claude, AU - Garcia,Luis, AU - Danos,Olivier, Y1 - 2004/11/04/ PY - 2004/11/6/pubmed PY - 2004/12/24/medline PY - 2004/11/6/entrez SP - 1796 EP - 9 JF - Science (New York, N.Y.) JO - Science VL - 306 IS - 5702 N2 - 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. SN - 1095-9203 UR - https://www.unboundmedicine.com/medline/citation/15528407/Rescue_of_dystrophic_muscle_through_U7_snRNA_mediated_exon_skipping_ L2 - https://www.sciencemag.org/cgi/pmidlookup?view=long&pmid=15528407 DB - PRIME DP - Unbound Medicine ER -