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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Shranjeno v:
Bibliografske podrobnosti
izdano v:Mol Ther
Main Authors: Xu, Li, Park, Ki Ho, Zhao, Lixia, Xu, Jing, El Refaey, Mona, Gao, Yandi, Zhu, Hua, Ma, Jianjie, Han, Renzhi
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2016
Teme:
Original Article
Online dostop: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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