Therapeutic development for Canavan disease using patient iPSCs introduced with the wild-type ASPA gene

Chao, JF; Feng, LZ; Ye, P; Chen, XW; Cui, Q; Sun, GH; Zhou, T; Tian, E; Li, WD; Hu, WD; Riggs, AD; Matalon, R; Shi, YH

Shi, YH (通讯作者),Beckman Res Inst City Hope, Div Stem Cell Biol Res, Dept Stem Cell Biol & Regenerat Med, 1500 E Duarte Rd, Duarte, CA 91010 USA.

ISCIENCE, 2022; 25 (6):