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

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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Bibliographic Details
Main Authors: Xi Xiang, Xiaoying Zhao, Xiaoguang Pan, Zhanying Dong, Jiaying Yu, Siyuan Li, Xue Liang, Peng Han, Kunli Qu, Jonas Brorson Jensen, Jean Farup, Fei Wang, Trine Skov Petersen, Lars Bolund, Huajing Teng, Lin Lin, Yonglun Luo
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
CRISPR
Cas9
gene therapy
Duchenne muscular dystrophy
reprogramming
regenerative medicine
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121000767

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http://www.sciencedirect.com/science/article/pii/S2162253121000767

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