Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice.
Li JJ, Lin X, Tang C, Lu YQ, Hu X, Zuo E, Li H, Ying W, Sun Y, Lai LL, Chen HZ, Guo XX, Zhang QJ, Wu S, Zhou C, Shen X, Wang Q, Lin MT, Ma LX, Wang N, Krainer AR, Shi L, Yang H, Chen WJ.
Li JJ, et al. Among authors: ma lx.
Natl Sci Rev. 2019 Sep 3;7(1):92-101. doi: 10.1093/nsr/nwz131. eCollection 2020 Jan.
Natl Sci Rev. 2019.
PMID: 34691481
Free PMC article.