Department of Molecular and Cellular Biology, Harvard University, Cambridge, Massachusetts, USA.
Nat Neurosci. 2013 Jul;16(7):780-9. doi: 10.1038/nn.3425. Epub 2013 Jun 25.
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.
人类神经退行性疾病是最难研究的疾病之一。特别是，由于无法轻易获得与这些疾病最相关的错误细胞类型，几十年来研究进展一直受阻。多能干细胞技术的最新进展使人们能够获得大量带有或不带有易感性突变的相关疾病神经元。虽然这一系列技术彻底改变了“体外疾病建模”领域，但如果要取得稳健、持久的发现，就必须谨慎使用这些技术。在这里，我们回顾了我们认为在使用干细胞研究神经疾病时存在的几个障碍，并提出了克服这些障碍的策略。
