《生命科学》 2016, 28(8): 902-906

人胚胎干细胞来源多巴胺前体细胞治疗帕金森病猴

王昱凯，周　琪*，胡宝洋*

(中国科学院动物研究所，北京 100101)

摘　要：摘　要：帕金森病(Parkinson’s disease， PD) 是一种常见于中老年人的神经系统退行性疾病，由中脑腹侧的多巴胺能(dopaminergic， DA) 神经元缺失造成。这类疾病可通过移植由人胚胎干细胞(human embryonic stem cells， hESC) 或其他途径获得的多巴胺能神经元实现治疗。然而，在应用于临床之前，需要对这些多巴胺能 神经元的安全性和有效性在合适的动物模型中进行充分、全面的评价。为评价由临床级人胚胎干细胞分化的多巴胺能神经元是否安全、有效，根据先导专项部署，我们建立了MPTP 诱导的帕金森病猴模型，并将由人胚胎干细胞定向分化的多巴胺能神经元植入受损猴脑区。结果表明，在所有接受细胞移植的猴中，未发生移植细胞形成的肿瘤和继发肿瘤。移植细胞可分化为多巴胺能神经元并使局部多巴胺水平提高，带来不同程度的行为学改善。这些发现提示，多能干细胞分化的多巴胺能神经元移植治疗帕金森病安全、有效，可进一步应用于帕金森病的治疗。

Long-term evaluation of human ESC-derived dopaminergic progenitor in monkey models of Parkinson’s disease

WANG Yu-Kai， ZHOU Qi*， HU Bao-Yang*

(Institute of Zoology， Chinese Academy of Sciences， Beijing 100101， China)

Abstract: Abstract: Parkinson’s disease (PD) is a progressive movement disorder common in elderly caused by loss of dopaminergic (DA) neurons in the ventral mesencephalon. Because of its nature of cell degeneration， PD is fairly suitable for cell replacement therapy， using DA neurons or progenitors from various sources， such as differentiation of hESCs. Yet， these hESC-derived progenies should be systematically assessed for safety and efficacy before
    transplantation into patients. Using MPTP-induced monkey models of Parkinson’s disease， we transplanted hESC-derived DA progenitors into monkey brains for long-term evaluation. We found all monkeys that received cell transplantation exhibited variable but discernible behavioral improvement without tumor formation from graft cell，indicating that hESCs are reliable cell sources for treating neural degenerative diseases such as PDs.