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journal article·2020-12Open access

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

Lizhao Feng, Jianfei Chao, E Tian, Li Li, Peng Ye, Mi Zhang, Xianwei Chen, Qi Cui, Guihua Sun, Tao Zhou, Gerardo Felix, Yue Qin, Wendong Li, Edward David Meza, Jeremy Klein, Lucy Ghoda, Weidong Hu, Yonglun Luo, Wei Dang, David Hsu, Joseph Gold, Steven A. Goldman, Reuben Matalon, Yanhong Shi

Advanced Science· Volume 7· Issue 23
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Source 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] [Image: see text]
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Research context

OpenAlex topics: Tissue Engineering and Regenerative Medicine

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