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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Библиографические подробности
Опубликовано в: :Mol Ther
Главные авторы: Xu, Li, Park, Ki Ho, Zhao, Lixia, Xu, Jing, El Refaey, Mona, Gao, Yandi, Zhu, Hua, Ma, Jianjie, Han, Renzhi
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2016
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Original Article
Online-ссылка: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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