人类诱导多能干细胞在心脏病药物研发中的应用模型。
Human iPSC modeling of heart disease for drug development.
机构信息
Stanford Cardiovascular Institute, 240 Pasteur Drive, Biomedical Innovation Building, Palo Alto, CA 94305, USA; Department of Medicine, Stanford University, Stanford, CA 94305, USA.
Stanford Cardiovascular Institute, 240 Pasteur Drive, Biomedical Innovation Building, Palo Alto, CA 94305, USA; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
出版信息
Cell Chem Biol. 2021 Mar 18;28(3):271-282. doi: 10.1016/j.chembiol.2021.02.016.
Human induced pluripotent stem cells (hiPSCs) have emerged as a promising platform for pharmacogenomics and drug development. In cardiology, they make it possible to produce unlimited numbers of patient-specific human cells that reproduce hallmark features of heart disease in the culture dish. Their potential applications include the discovery of mechanism-specific therapeutics, the evaluation of safety and efficacy in a human context before a drug candidate reaches patients, and the stratification of patients for clinical trials. Although this new technology has the potential to revolutionize drug discovery, translational hurdles have hindered its widespread adoption for pharmaceutical development. Here we discuss recent progress in overcoming these hurdles that should facilitate the use of hiPSCs to develop new medicines and individualize therapies for heart disease.
人类诱导多能干细胞（hiPSCs）已成为药物基因组学和药物开发的有前途的平台。在心脏病学中，它们使得能够在培养皿中产生无限数量的患者特异性人类细胞，这些细胞再现心脏病的标志性特征。它们的潜在应用包括发现机制特异性治疗药物，在药物候选物到达患者之前在人类环境中评估安全性和有效性，以及对临床试验患者进行分层。尽管这项新技术有可能彻底改变药物发现，但转化障碍阻碍了其在药物开发中的广泛应用。在这里，我们讨论了克服这些障碍的最新进展，这些进展应该有助于利用 hiPSCs 开发新药和为心脏病患者进行个体化治疗。
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