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CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by genetic mutations that lead to the disruption of dystrophin in muscle fibers. There is no curative treatment for this devastating disease. Clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) has emer...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Xu, Li, Park, Ki Ho, Zhao, Lixia, Xu, Jing, El Refaey, Mona, Gao, Yandi, Zhu, Hua, Ma, Jianjie, Han, Renzhi
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4786912/
https://ncbi.nlm.nih.gov/pubmed/26449883
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.192
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