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

• Main Authors: Xi Xiang (Author), Xiaoying Zhao (Author), Xiaoguang Pan (Author), Zhanying Dong (Author), Jiaying Yu (Author), Siyuan Li (Author), Xue Liang (Author), Peng Han (Author), Kunli Qu (Author), Jonas Brorson Jensen (Author), Jean Farup (Author), Fei Wang (Author), Trine Skov Petersen (Author), Lars Bolund (Author), Huajing Teng (Author), Lin Lin (Author), Yonglun Luo (Author)
• Format: Book
• Published: Elsevier, 2021-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...