Gladstone Institutes , 1650 Owens Street, San Francisco, California 94158, United States.
J Med Chem. 2016 Jan 14;59(1):2-15. doi: 10.1021/acs.jmedchem.5b00789. Epub 2015 Sep 4.
In vitro cell models are invaluable tools for studying diseases and discovering drugs. Human induced pluripotent stem cells, particularly derived from patients, are an advantageous resource for generating ample supplies of cells to create unique platforms that model disease. This manuscript will review recent developments in modeling a variety of diseases (including their cellular phenotypes) with induced pluripotent stem cells derived from patients. It will also describe how researchers have exploited these models to validate drugs as potential therapeutics for these devastating diseases.
体外细胞模型是研究疾病和发现药物的宝贵工具。人类诱导多能干细胞，特别是从患者中获得的诱导多能干细胞，是产生大量细胞的有利资源，可创建独特的平台来模拟疾病。本文将综述利用患者来源的诱导多能干细胞对多种疾病（包括其细胞表型）进行建模的最新进展。本文还将描述研究人员如何利用这些模型来验证药物作为这些破坏性疾病潜在治疗方法的有效性。
