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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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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Full Papers
On-line přístup: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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