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Molecular Therapeutic Strategies Targeting Duchenne Muscular Dystrophy

Since discovery of the gene for Duchenne muscular dystrophy more than 20 years ago, scientists have worked to apply molecular principles for restoration of the dystrophin protein and correction of the underlying physiologic defect that predisposes muscle fibers to injury. Recent studies provide real...

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Autors principals: Mendell, Jerry R., Rodino-Klapac, Louise R., Malik, Vinod
Format: Artigo
Idioma:Inglês
Publicat: 2010
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3674570/
https://ncbi.nlm.nih.gov/pubmed/20498331
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073810371005
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