Disruption of splicing-regulatory elements using CRISPR/Cas9 rescues spinal muscular atrophy in human iPSCs and mice
Li, Jin-Jing, Lin, Xiang, Tang, Cheng, Lu, Ying-Qian, Hu, Xinde, Zuo, Erwei, Li, He, Ying, Wenqin, Sun, Yidi, Lai, Lu-Lu, Chen, Hai-Zhu, Guo, Xin-Xin, Zhang, Qi-Jie, Wu, Shuang, Zhou, Changyang, Shen,Language:
english
Journal:
National Science Review
DOI:
10.1093/nsr/nwz131
Date:
September, 2019
File:
PDF, 1.08 MB
english, 2019