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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Adv Sci (Weinh)
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: John Wiley and Sons Inc. 2020
Pynciau:
Full Papers
Mynediad Ar-lein: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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