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用于帕金森病建模与治疗的干细胞

Stem Cells for Modeling and Therapy of Parkinson's Disease.

作者信息

Zhang Qingxi, Chen Wanling, Tan Sheng, Lin Tongxiang

机构信息

1 Center for Regenerative and Translational Medicine, The Second Affiliated Hospital, Guangzhou University of Chinese Medicine (Guangdong Provincial Hospital of Chinese Medicine) , Guangzhou, China .

2 Department of Neurology, Zhujiang Hospital of Southern Medical University , Guangzhou, China .

出版信息

Hum Gene Ther. 2017 Jan;28(1):85-98. doi: 10.1089/hum.2016.116. Epub 2016 Sep 26.

Abstract

Parkinson's disease (PD) is the second most frequent neurodegenerative disease after Alzheimer's disease, which is characterized by a low level of dopamine being expressing in the striatum and a deterioration of dopaminergic neurons (DAn) in the substantia nigra pars compacta. Generation of PD-derived DAn, including differentiation of human embryonic stem cells, human neural stem cells, human-induced pluripotent stem cells, and direct reprogramming, provides an ideal tool to model PD, creating the possibility of mimicking key essential pathological processes and charactering single-cell changes in vitro. Furthermore, thanks to the understanding of molecular neuropathogenesis of PD and new advances in stem-cell technology, it is anticipated that optimal functionally transplanted DAn with targeted correction and transgene-free insertion will be generated for use in cell transplantation. This review elucidates stem-cell technology for modeling PD and offering desired safe cell resources for cell transplantation therapy.

摘要

帕金森病(PD)是仅次于阿尔茨海默病的第二常见神经退行性疾病,其特征是纹状体中多巴胺表达水平低以及黑质致密部多巴胺能神经元(DAn)退化。源自帕金森病的多巴胺能神经元的生成,包括人类胚胎干细胞、人类神经干细胞、人类诱导多能干细胞的分化以及直接重编程,为帕金森病建模提供了理想工具,使得在体外模拟关键的基本病理过程以及表征单细胞变化成为可能。此外,由于对帕金森病分子神经发病机制的理解以及干细胞技术的新进展,预计将生成具有靶向校正和无转基因插入的最佳功能的移植多巴胺能神经元,用于细胞移植。本综述阐述了用于帕金森病建模以及为细胞移植治疗提供所需安全细胞资源的干细胞技术。

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