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In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing one or more exons from the mutated transcript can produce an in-frame mRNA and a truncated but still functional protein. In thi...
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Publicado no: | Science |
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Main Authors: | , , , , , , , , , , , , , , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
2015
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Assuntos: | |
Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4924477/ https://ncbi.nlm.nih.gov/pubmed/26721686 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/science.aad5177 |
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