Buck Institute for Research on Aging, 8001 Redwood Boulevard, Novato, CA 94945, USA.
Stem Cell Res Ther. 2011 Jul 28;2(4):32. doi: 10.1186/scrt73.
Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, and Friedreich's ataxia are the most common human neurodegenerative diseases pathologically characterized by a progressive and specific loss of certain neuronal populations. The exact mechanisms of neuronal cell death in these diseases are unclear, although some forms of the diseases are inherited and genes causing these diseases have been identified. Currently there are no effective clinical therapies for many of these diseases. The recently acquired ability to reprogram human adult somatic cells to induced pluripotent stem cells (iPSCs) in culture may provide a powerful tool for in vitro neurodegenerative disease modeling and an unlimited source for cell replacement therapy. In the present review, we summarize recent progress on iPSC generation and differentiation into neuronal cell types and discuss the potential application for in vitro disease mechanism study and in vivo cell replacement therapy.
阿尔茨海默病、帕金森病、亨廷顿病、肌萎缩侧索硬化症和弗里德里希共济失调是最常见的人类神经退行性疾病，其病理特征为某些神经元群体进行性和特异性丧失。尽管某些形式的疾病是遗传性的，并且已经确定了导致这些疾病的基因，但这些疾病中神经元细胞死亡的确切机制尚不清楚。目前，许多此类疾病都没有有效的临床治疗方法。最近获得的在培养中将人类成体细胞重编程为诱导多能干细胞 (iPSC) 的能力可能为体外神经退行性疾病建模提供强大工具，并为细胞替代疗法提供无限的来源。在本综述中，我们总结了 iPSC 生成和分化为神经元细胞类型的最新进展，并讨论了其在体外疾病机制研究和体内细胞替代治疗中的潜在应用。
