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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: | |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
2010
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Assuntos: | |
Acesso em linha: | 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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