Morizane Asuka, Takahashi Jun
Department of Cell Growth and Differentiation, Kyoto University, Japan.
Brain Nerve. 2012 Jan;64(1):29-37.
Parkinson's disease has been so far commonly treated with medication therapy. Although the medication works effectively in the initial phase, it turns out to be less effective at the later stage of the disease. Recently, induced pluripotent stem (iPS) cells have attracted much attention because of their potential to cure diseases such as Parkinson's disease. Due to the accumulating clinical experiences of cell transplantation procedures with aborted fetal tissues, Parkinson's disease has become one of the most promising targets for the clinical application of this iPS cell technology. In this review, we will summarize the ongoing research in the field of iPS cells and Parkinson's disease. The method for establishing iPS cells has advanced rapidly that can be applied in the clinical stage in terms of avoiding the use of viral vectors, xenogenic materials, etc. The differentiation protocol to derive the dopamine neurons from iPS cells has also been improved. However, several issues, such as the risk of tumor formation and the poor survival of the grafted dopamine neurons in vivo remain to be solved before these cells can be used in the clinical settings. Other than cell transplantations, iPS cell technology can also provide a valuable platform for disease analysis and drug development with in vitro systems of human cells. Several lines of iPS cells have already been established from Parkinson's disease patients with either sporadic or genetic background. For patients to achieve maximum benefits of this technology, further research must be conducted in both fields, that is, cell transplantation and the disease modeling with patient-derived iPS cells.
迄今为止，帕金森病通常采用药物治疗。尽管药物在疾病初期疗效显著，但在疾病后期效果却不尽人意。近来，诱导多能干细胞（iPS细胞）因其治愈帕金森病等疾病的潜力而备受关注。鉴于利用流产胎儿组织进行细胞移植手术的临床经验不断积累，帕金森病已成为iPS细胞技术临床应用最具前景的目标之一。在本综述中，我们将总结iPS细胞与帕金森病领域的现有研究。iPS细胞的建立方法发展迅速，在避免使用病毒载体、异种材料等方面已可应用于临床阶段。从iPS细胞诱导分化生成多巴胺能神经元的方案也得到了改进。然而，在这些细胞能够用于临床之前，仍有几个问题有待解决，比如肿瘤形成风险以及移植的多巴胺能神经元在体内存活率低等问题。除了细胞移植，iPS细胞技术还可为利用人类细胞体外系统进行疾病分析和药物研发提供一个有价值的平台。已经从散发性或遗传性帕金森病患者中建立了多株iPS细胞系。为使患者从这项技术中获得最大益处，必须在细胞移植和利用患者来源的iPS细胞进行疾病建模这两个领域开展进一步研究。
