Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45

OBJECTIVE: Exon-skipping therapies aim to convert Duchenne muscular dystrophy (DMD) into less severe Becker muscular dystrophy (BMD) by altering pre-mRNA splicing to restore an open reading frame, allowing translation of an internally deleted and partially functional dystrophin protein. The most com...

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Publicado no:Ann Neurol
Main Authors: Findlay, Andrew R., Wein, Nicolas, Kaminoh, Yuuki, Taylor, Laura E., Dunn, Diane M., Mendell, Jerry R., King, Wendy M., Pestronk, Alan, Florence, Julaine M., Mathews, Katherine D., Finkel, Richard S., Swoboda, Kathryn J., Howard, Michael T., Day, John W., McDonald, Craig, Nicolas, Aurélie, Le Rumeur, Elisabeth, Weiss, Robert B., Flanigan, Kevin M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4376581/
https://ncbi.nlm.nih.gov/pubmed/25612243
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24365
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