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

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 dise...

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Vydáno v:Adv Sci (Weinh)
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2020
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Inside Back Cover
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7709969/
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/advs.202070131
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