Laboratory of Genetics, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, La Jolla, CA, 92037, USA.
Neurobiology Section, Division of Biological Sciences, University of California San Diego, La Jolla, CA, 92093, USA.
Protein Cell. 2020 Jan;11(1):45-59. doi: 10.1007/s13238-019-0638-8. Epub 2019 May 27.
Neuropsychiatric disorders are complex disorders characterized by heterogeneous genetic variations, variable symptoms, and widespread changes in anatomical pathology. In the context of neuropsychiatric disorders, limited access to relevant tissue types presents challenges for understanding disease etiology and developing effective treatments. Induced pluripotent stem cells (iPSCs) reprogrammed from patient somatic cells offer an opportunity to recapitulate disease development in relevant cell types, and they provide novel approaches for understanding disease mechanisms and for development of effective treatments. Here we review recent progress and challenges in differentiation paradigms for generating disease-relevant cells and recent studies of neuropsychiatric disorders using human pluripotent stem cell (hPSC) models where cellular phenotypes linked to disease have been reported. The use of iPSC-based disease models holds great promise for understanding disease mechanisms and supporting discovery of effective treatments.
神经精神疾病是一类复杂的疾病,其特征为具有异质性的遗传变异、多变的症状和广泛的解剖病理学变化。在神经精神疾病的背景下,相关组织类型的获取受限,这给理解疾病病因和开发有效疗法带来了挑战。由患者体细胞重编程而来的诱导多能干细胞(iPSC)为在相关细胞类型中重现疾病发展提供了机会,并且为理解疾病机制和开发有效疗法提供了新的方法。本文综述了用于生成疾病相关细胞的分化范例方面的最新进展和挑战,以及使用人类多能干细胞(hPSC)模型来研究神经精神疾病的最新研究进展,其中报告了与疾病相关的细胞表型。基于 iPSC 的疾病模型的使用为理解疾病机制和支持有效疗法的发现带来了巨大的希望。