人类诱导多能干细胞中的神经退行性变表型分析。
Phenotyping Neurodegeneration in Human iPSCs.
机构信息
Computational and Systems Biology, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA; email:
Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.
出版信息
Annu Rev Biomed Data Sci. 2021 Jul 20;4:83-100. doi: 10.1146/annurev-biodatasci-092820-025214. Epub 2021 Apr 23.
Abstract
Induced pluripotent stem cell (iPSC) technology holds promise for modeling neurodegenerative diseases. Traditional approaches for disease modeling using animal and cellular models require knowledge of disease mutations. However, many patients with neurodegenerative diseases do not have a known genetic cause. iPSCs offer a way to generate patient-specific models and study pathways of dysfunction in an in vitro setting in order to understand the causes and subtypes of neurodegeneration. Furthermore, iPSC-based models can be used to search for candidate therapeutics using high-throughput screening. Here we review how iPSC-based models are currently being used to further our understanding of neurodegenerative diseases, as well as discuss their challenges and future directions.
摘要
诱导多能干细胞（iPSC）技术有望用于神经退行性疾病的建模。传统的使用动物和细胞模型进行疾病建模的方法需要了解疾病突变。然而，许多神经退行性疾病患者没有已知的遗传原因。iPSC 提供了一种在体外环境中生成患者特异性模型并研究功能障碍途径的方法，以了解神经退行性变的原因和亚型。此外，基于 iPSC 的模型可用于使用高通量筛选寻找候选治疗药物。在这里，我们回顾了基于 iPSC 的模型如何用于进一步了解神经退行性疾病，并讨论了它们的挑战和未来方向。
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