Efficient correction of Duchenne muscular dystrophy mutations by SpCas9 and dual gRNAs
Xi Xiang, Xiaoying Zhao, Xiaoguang Pan, Zhanying Dong, Jiaying Yu, Siyuan Li, Xue Liang, Peng Han, Kunli Qu, Jonas Jensen, Jean Farup, Fei Wang, Trine Skov Petersen, Lars Bolund, Huajing Teng, Lin Lin, Yonglun Luo
Abstract
expression at both the mRNA and protein levels was confirmed in the induced myotubes. With further development, the combination of SpCas9-dual gRNA-corrected DMD patient fibroblasts and transdifferentiation may provide a valuable therapeutic strategy for DMD.
Topics & Concepts
Duchenne muscular dystrophyDual (grammatical number)Muscular dystrophyGeneticsBiologyPhilosophyLinguisticsCRISPR and Genetic EngineeringMuscle Physiology and DisordersRNA regulation and disease