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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Dades bibliogràfiques
Publicat a:Adv Sci (Weinh)
Autors principals: 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
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2020
Matèries:
Full Papers
Accés en línia: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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