摘要
帕金森病是一类以黑质多巴胺能神经元的进行性变性为特点的运动性疾病，在以往对帕金森病的研究巾，由于缺乏对患者特异性的多巴胺能神经元的认识，阻碍了人们对疾病机制的认识和药物的研发。诱导性多潜能干细胞的出现弥补了这一缺陷，将人们对帕金森患者黑质多巴胺能神经元易损性和疾病的治疗研究推进到一个更崭新的发展阶段。
Parkinson＇s disease （PD） is a movement disorder characterized by a relatively selective degeneration of nigral dopaminergic （DA） neurons and a core set of locomotor symptoms including rest tremor, rigidity and bradykinesia. One of the greatest obstacles for PD research is the lack of live patient-specific nigral DA neurons for mechanistic studies and drug disco- very research. The discovery of induced pluripotent stem cells （iPSCs） has made this possible. It is transforming PD research towards the use of the human model system. It will allow us to identify the unique vulnerahilities of human midbrain DA neu- rons and ultimately produce a disease-modifying therapy based on mechanistic understanding of PD.
出处
《转化医学研究（电子版）》
2013年第1期23-36,共14页
Translational Medicine Research(Electronic Edition)
