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Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA

Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. DMD is characterized by progressive muscle weakness and a shortened life span, and there is no effective treatment. We used clustered...

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Détails bibliographiques
Publié dans:Science
Auteurs principaux: Long, Chengzu, McAnally, John R., Shelton, John M., Mireault, Alex A., Bassel-Duby, Rhonda, Olson, Eric N.
Format: Artigo
Langue:Inglês
Publié: 2014
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Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4398027/
https://ncbi.nlm.nih.gov/pubmed/25123483
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/science.1254445
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