Translational Biology, Research and Early Development, Biogen, 115 Broadway, Cambridge, MA 02142, USA.
Translational Biology, Research and Early Development, Biogen, 115 Broadway, Cambridge, MA 02142, USA.
Neuron. 2018 Nov 21;100(4):783-797. doi: 10.1016/j.neuron.2018.10.033.
From the beginning, induced pluripotent stem cell (iPSC) technology was touted as a path to improve our understanding of disease biology and enable drug discovery. Advances in iPSC culture, genome engineering, and differentiation protocols have rapidly expanded the use of iPSC-derived disease models from the specialized work of stem cell biology into the mainstream toolkit of cellular neuroscience. Here we provide guidance for using iPSC-derived neurons for disease modeling with a focus on enabling screening platforms amenable to therapeutic drug discovery. We also highlight the potential for incorporating three-dimensional systems that may create more translational in vitro models.
从一开始，诱导多能干细胞（iPSC）技术就被誉为一种能够增进我们对疾病生物学理解并推动药物研发的途径。iPSC 培养、基因组编辑和分化方案的进展，迅速将 iPSC 衍生疾病模型的应用从干细胞生物学的专业领域扩展到细胞神经科学的主流工具包中。在这里，我们提供了使用 iPSC 衍生神经元进行疾病建模的指导，重点是构建适合治疗药物发现的筛选平台。我们还强调了整合可能创建更具转化能力的体外模型的三维系统的潜力。
