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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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| Publicado no: | Science |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2014
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| Assuntos: | |
| Acesso em linha: | 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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