Stem Cell Therapy: Cell‐Based Therapy for Canavan Disease Using Human iPSC‐Derived NPCs and OPCs (Adv. Sci. 23/2020). Issue 23 (2nd December 2020)

• Record Type: Journal Article
• Authors: Feng, Lizhao; Chao, Jianfei; Tian, E; Li, Li; Ye, Peng; Zhang, Mi; Chen, Xianwei; Cui, Qi; Sun, Guihua; Zhou, Tao; Felix, Gerardo; Qin, Yue; Li, Wendong; Meza, Edward David; Klein, Jeremy; Ghoda, Lucy; Hu, Weidong; Luo, Yonglun; Dang, Wei; Hsu, David; Gold, Joseph; Goldman, Steven A.; Matalon, Reuben; Shi, Yanhong
• Is Part Of: Advanced science. Volume 7:Issue 23(2020)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2020-12-02
• Subjects: Science -- Periodicals; 505
• DOI: 10.1002/advs.202070131
• Languages: English
• ISSNs: 2198-3844

Abstract

Abstract : Canavan disease (CD) is a leukodystrophy caused by aspartoacylase (ASPA) gene mutation with no cure so far. In this study, a functional ASPA was introduced into patient iPSC‐derived neural progenitor cells (iNPCs) and the engineered ASPA iNPCs could rescue major pathological features of CD in a disease mouse model. These patient iPSC‐derived cellular products are promising cell therapies for CD patients. [Image designed and prepared by Olivia Sun]