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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Publicat a:Mol Ther
Autors principals: Xu, Li, Park, Ki Ho, Zhao, Lixia, Xu, Jing, El Refaey, Mona, Gao, Yandi, Zhu, Hua, Ma, Jianjie, Han, Renzhi
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2016
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Original Article
Accés en línia: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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