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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther
Egile Nagusiak: Xu, Li, Park, Ki Ho, Zhao, Lixia, Xu, Jing, El Refaey, Mona, Gao, Yandi, Zhu, Hua, Ma, Jianjie, Han, Renzhi
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Nature Publishing Group 2016
Gaiak:
Original Article
Sarrera elektronikoa: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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