Efficient correction of Duchenne muscular dystrophy mutations by SpCas9 and dual gRNAs

CRISPR gene therapy is one promising approach for treatment of Duchenne muscular dystrophy (DMD), which is caused by a large spectrum of mutations in the dystrophin gene. To broaden CRISPR gene editing strategies for DMD treatment, we report the efficient restoration of dystrophin expression in indu...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Nucleic Acids
Prif Awduron: Xiang, Xi, Zhao, Xiaoying, Pan, Xiaoguang, Dong, Zhanying, Yu, Jiaying, Li, Siyuan, Liang, Xue, Han, Peng, Qu, Kunli, Jensen, Jonas Brorson, Farup, Jean, Wang, Fei, Petersen, Trine Skov, Bolund, Lars, Teng, Huajing, Lin, Lin, Luo, Yonglun
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2021
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC8039775/
https://ncbi.nlm.nih.gov/pubmed/33868784
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2021.03.005
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