Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle: Progress in Exon Skipping and Stop Codon Read Through

The identification of the Duchenne muscular dystrophy gene and protein in the late 1980s led to high hopes of rapid translation to molecular therapeutics. These hopes were fueled by early reports of delivering new functional genes to dystrophic muscle in mouse models using gene therapy and stem cell...

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Detalles Bibliográficos
Autores principales: Hoffman, Eric P., Bronson, Abby, Levin, Arthur A., Takeda, Shin'ichi, Yokota, Toshifumi, Baudy, Andreas R., Connor, Edward M.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society for Investigative Pathology 2011
Materias:
Review
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3124804/
https://ncbi.nlm.nih.gov/pubmed/21703390
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajpath.2011.03.050
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