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心脏疾病的多能干细胞模型及其对药物发现和开发的意义。

Pluripotent stem cell models of cardiac disease and their implication for drug discovery and development.

机构信息

Department of Anatomy and Embryology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands.

出版信息

Trends Mol Med. 2011 Sep;17(9):475-84. doi: 10.1016/j.molmed.2011.05.001. Epub 2011 Jun 22.

DOI:10.1016/j.molmed.2011.05.001
PMID:21703926
Abstract

Recent advances in pluripotent stem cell biology now make it possible to generate human cardiomyocytes in vitro from both healthy individuals and from patients with cardiac abnormalities. This offers unprecedented opportunities to study cardiac disease development 'in a dish' and establish novel platforms for drug discovery, either to prevent disease progression or to reverse it. In this review paper, we discuss some of the genetic diseases that affect the heart and illustrate how these new paradigms could assist our understanding of cardiac pathogenesis and aid in drug discovery. In particular, we highlight the limitations of other commonly used model systems in predicting the consequences of drug exposure on the human heart.

摘要

最近多能干细胞生物学的进展使得人们有可能从健康个体和患有心脏异常的患者中在体外生成人心肌细胞。这为在“培养皿”中研究心脏疾病的发展提供了前所未有的机会,并为药物发现建立了新的平台,无论是预防疾病进展还是逆转疾病。在这篇综述论文中,我们讨论了一些影响心脏的遗传性疾病,并说明了这些新范例如何帮助我们理解心脏发病机制并辅助药物发现。特别是,我们强调了其他常用模型系统在预测药物暴露对人类心脏的影响方面的局限性。

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