Denning Chris, Borgdorff Viola, Crutchley James, Firth Karl S A, George Vinoj, Kalra Spandan, Kondrashov Alexander, Hoang Minh Duc, Mosqueira Diogo, Patel Asha, Prodanov Ljupcho, Rajamohan Divya, Skarnes William C, Smith James G W, Young Lorraine E
Department of Stem Cell Biology, Centre for Biomolecular Sciences, University of Nottingham, NG7 2RD, United Kingdom.
Department of Stem Cell Biology, Centre for Biomolecular Sciences, University of Nottingham, NG7 2RD, United Kingdom.
Biochim Biophys Acta. 2016 Jul;1863(7 Pt B):1728-48. doi: 10.1016/j.bbamcr.2015.10.014. Epub 2015 Oct 31.
Cardiomyocytes from human pluripotent stem cells (hPSCs-CMs) could revolutionise biomedicine. Global burden of heart failure will soon reach USD $90bn, while unexpected cardiotoxicity underlies 28% of drug withdrawals. Advances in hPSC isolation, Cas9/CRISPR genome engineering and hPSC-CM differentiation have improved patient care, progressed drugs to clinic and opened a new era in safety pharmacology. Nevertheless, predictive cardiotoxicity using hPSC-CMs contrasts from failure to almost total success. Since this likely relates to cell immaturity, efforts are underway to use biochemical and biophysical cues to improve many of the ~30 structural and functional properties of hPSC-CMs towards those seen in adult CMs. Other developments needed for widespread hPSC-CM utility include subtype specification, cost reduction of large scale differentiation and elimination of the phenotyping bottleneck. This review will consider these factors in the evolution of hPSC-CM technologies, as well as their integration into high content industrial platforms that assess structure, mitochondrial function, electrophysiology, calcium transients and contractility. This article is part of a Special Issue entitled: Cardiomyocyte Biology: Integration of Developmental and Environmental Cues in the Heart edited by Marcus Schaub and Hughes Abriel.
来自人类多能干细胞的心肌细胞(hPSC-CMs)可能会给生物医学带来变革。心力衰竭的全球负担很快将达到900亿美元,而意外的心脏毒性是28%的药物撤市的原因。hPSC分离、Cas9/CRISPR基因组工程以及hPSC-CM分化方面的进展改善了患者护理,推动药物进入临床,并开启了安全药理学的新时代。然而,使用hPSC-CMs进行心脏毒性预测的结果却从失败到几乎完全成功各不相同。由于这可能与细胞不成熟有关,人们正在努力利用生化和生物物理线索,使hPSC-CMs约30种结构和功能特性中的许多特性向成年心肌细胞的特性发展。hPSC-CM广泛应用所需的其他进展包括亚型特异性、大规模分化成本降低以及消除表型分析瓶颈。本综述将探讨这些因素在hPSC-CM技术发展中的作用,以及它们如何融入评估结构、线粒体功能、电生理学、钙瞬变和收缩性的高内涵工业平台。本文是由Marcus Schaub和Hughes Abriel编辑的名为《心肌细胞生物学:心脏发育和环境线索的整合》特刊的一部分。