Cell‐Based Therapy for Canavan Disease Using Human iPSC‐Derived NPCs and OPCs

Canavan disease (CD) is a fatal leukodystrophy caused by mutation of the aspartoacylase (ASPA) gene, which leads to deficiency in ASPA activity, accumulation of the substrate N‐acetyl‐L‐aspartate (NAA), demyelination, and spongy degeneration of the brain. There is neither a cure nor a standard treat...

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Bibliografiske detaljer
Udgivet i:Adv Sci (Weinh)
Main 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
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
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Full Papers
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7709977/
https://ncbi.nlm.nih.gov/pubmed/33304759
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/advs.202002155
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