Human iPSC modeling of heart disease for drug development.

Human iPSC modeling of heart disease for drug development.
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DOI:
10.1016/j.chembiol.2021.02.016
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发表时间:
2021-03-18
影响因子:
8.6
通讯作者:
Mercola M
Mercola M
中科院分区:
生物学1区
文献类型:
--
作者:
Hnatiuk AP;Briganti F;Staudt DW;Mercola M

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人诱导多能干细胞(HiPSCs)已成为药物基因组学和药物开发的重要平台。在心脏病学方面,它们使在培养皿中产生无限数量的特定于患者的人类细胞成为可能,这些细胞可以复制心脏病的标志性特征。它们的潜在应用包括发现特定机制的疗法,在候选药物到达患者之前在人体环境中评估安全性和有效性,以及对患者进行临床试验的分层。尽管这项新技术有可能给药物发现带来革命性的变化,但翻译上的障碍阻碍了它在制药开发中的广泛采用。在这里,我们讨论在克服这些障碍方面的最新进展,这些障碍应该有助于使用HiPSCs来开发新药和个体化治疗心脏病。人类诱导多能干细胞(HiPSC)技术的使用可能会给心血管药物的发现带来革命性的变化。Hnatiuk等人。回顾最初的几项大规模研究,讨论对采用这项技术很重要的经验教训。
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. The use of human induced pluripotent stem cell (hiPSC) technology could revolutionize cardiovascular drug discovery. Hnatiuk et al. review the first few large scale studies and discuss the lessons learned that are important for adopting this technology.
IPSC衍生的心肌细胞的高通量生理筛查用于药物开发。
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