Kevin Eggan2014-02-27 11:57 AM

Opportunities and challenges of pluripotent stem cell neurodegenerative disease models. 多能干细胞神经退行性疾病模型的机遇与挑战 

Abstract

Human neurodegenerative disorders are among the most difficult to study. In particular, the inability to readily obtain the faulty cell types most relevant to these diseases has impeded progress for decades. Recent advances in pluripotent stem cell technology now grant access to substantial quantities of disease-pertinent neurons both with and without predisposing mutations. While this suite of technologies has revolutionized the field of 'in vitro disease modeling', great care must be taken in their deployment if robust, durable discoveries are to be made. Here we review what we perceive to be several of the stumbling blocks in the use of stem cells for the study of neurological disease and offer strategies to overcome them.

论文摘要

人类神经退行性疾病仍是人类研究难题之一。由于无法轻易获得与这些疾病最紧密相关的缺陷细胞类型,几十年来尤其阻碍着研究的进步。多能干细胞技术的最新进展使得我们可以大量得到易于和不易于诱发突变的相关疾病神经元。虽然这套技术彻底革新了“体外疾病模型”的研究领域,但如果想要进行有力持久的探索,我们仍需部署谨慎。我们在此综述了在神经系统疾病研究中所感知的一些对干细胞应用的阻碍且提供了克服阻碍的策略。 

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Kevin Eggan

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