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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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7709977/
https://www.ncbi.nlm.nih.gov/pubmed/33304759
http://dx.doi.org/10.1002/advs.202002155