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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.

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 , 306 (5702) pp. 1796-1799. 10.1126/science.1104297.

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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.

Type: Article
Title: Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.
Location: United States
DOI: 10.1126/science.1104297
Keywords: Animals, Dependovirus, Dystrophin, Exons, Genetic Therapy, Genetic Vectors, Introns, Mice, Mice, Inbred mdx, Muscle Contraction, Muscle Fibers, Skeletal, Muscle, Skeletal, Muscular Dystrophy, Animal, Muscular Dystrophy, Duchenne, Mutation, Oligonucleotides, Antisense, RNA Splicing, RNA, Messenger, RNA, Small Nuclear, Transfection
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: http://discovery.ucl.ac.uk/id/eprint/90974
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