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...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8039775/
https://www.ncbi.nlm.nih.gov/pubmed/33868784
http://dx.doi.org/10.1016/j.omtn.2021.03.005

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