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The Potential of Exon Skipping for Treatment for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is mainly caused by mutations that disrupt the generation of a translatable mRNA transcript. Most such mutations occur in parts of the gene that are not essential for its function and thus might be eliminated from the transcript to permit translation of a partially functi...

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Autor principal: Partridge, Terence
Format: Artigo
Idioma:Inglês
Publicat: 2010
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3695465/
https://ncbi.nlm.nih.gov/pubmed/20519674
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073810371130
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