Yerkes National Primate Research Center, 954 Gatewood Rd., N.E. Atlanta, GA 39329, USA.
J Genet Genomics. 2012 Jun 20;39(6):253-9. doi: 10.1016/j.jgg.2012.04.006. Epub 2012 May 9.
Pluripotent cellular models have shown great promise in the study of a number of neurological disorders. Several advantages of using a stem cell model include the potential for cells to derive disease relevant neuronal cell types, providing a system for researchers to monitor disease progression during neurogenesis, along with serving as a platform for drug discovery. A number of stem cell derived models have been employed to establish in vitro research models of Huntington's disease that can be used to investigate cellular pathology and screen for drug and cell-based therapies. Although some progress has been made, there are a number of challenges and limitations that must be overcome before the true potential of this research strategy is achieved. In this article we review current stem cell models that have been reported, as well as discuss the issues that impair these studies. We also highlight the prospective application of Huntington's disease stem cell models in the development of novel therapeutic strategies and advancement of personalized medicine.
多能细胞模型在研究多种神经退行性疾病方面显示出巨大的潜力。使用干细胞模型有几个优点，包括细胞有可能衍生出与疾病相关的神经元细胞类型，为研究人员在神经发生过程中监测疾病进展提供了一个系统，同时也为药物发现提供了一个平台。已经有许多干细胞衍生模型被用于建立亨廷顿病的体外研究模型，这些模型可用于研究细胞病理学，并筛选药物和细胞治疗方法。尽管已经取得了一些进展，但在真正实现这一研究策略的潜力之前，还需要克服许多挑战和限制。本文综述了目前报道的干细胞模型，并讨论了影响这些研究的问题。我们还强调了亨廷顿病干细胞模型在开发新型治疗策略和推进个性化医疗方面的应用前景。
