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人诱导多能干细胞在扩张型心肌病模型建立及应用中的研究进展

谢曼婷, 谢冰冰, 向秋玲^*

中山大学中山医学院，广州 510080

摘要

扩张型心肌病(dilated cardiomyopathy, DCM)是一种具有心肌结构和功能异常的非缺血性心肌病。利用DCM患者的心肌细胞进行细胞功能研究受伦理等多方面限制，相应的动物模型难以构建，疾病发生和发展的机制尚不清晰。人诱导多能干细胞(human induced pluripotent stem cells, hiPSCs)的出现为DCM的基础研究提供了新的方向。研究人员利用hiPSCs诱导分化获得心肌细胞(hiPSCs-derived cardiomyocytes, hiPSC-CMs)进行DCM疾病模型构建，利用该模型进行药物筛选，并进一步为DCM的病理机制探究及干预措施提供新的见解。本文对DCM患者特异性hiPSC-CMs (DCM-hiPSC-CMs)模型构建、药物筛选、机制研究等方面研究进展进行综述。

关键词： 扩张型心肌病; 诱导多能干细胞; 疾病模型; 研究进展

Research progress of human induced pluripotent stem cells in the establishment and application of dilated cardiomyopathy disease model 

XIE Man-Ting, XIE Bing-Bing, XIANG Qiu-Ling^*

Zhongshan School of Medicine, Sun Yat-sen University, Guangzhou 510080, China

Abstract

Dilated cardiomyopathy (DCM) is a non-ischemic cardiomyopathy with abnormal myocardial structure and function. It is challenging to construct human primary cardiac myocytes from DCM patients due to ethical constraints. In addition, animal models failed to adequately replicate the complexity of the human disease. The mechanism of DCM remains unclear. The emergence of human induced pluripotent stem cells (hiPSCs) provides a new tool for basic research in DCM. Researchers have produced hiPSCs-derived cardiomyocytes (hiPSC-CMs) and applied them to drug screening, leading to new insight into the pathomechanism and treatment in DCM. This review summarizes the research progress in the establishment, drug screening and mechanism research of DCM patient-specific hiPSC-CMs (DCM-hiPSC-CMs) model.

Key words: dilated cardiomyopathy; induced pluripotent stem cells; disease model; research progress

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通讯作者：向秋玲　　E-mail:

引用本文：

谢曼婷, 谢冰冰, 向秋玲. 人诱导多能干细胞在扩张型心肌病模型建立及应用中的研究进展[J]. 生理学报 2024; 76 (5): 775-782.

XIE Man-Ting, XIE Bing-Bing, XIANG Qiu-Ling. Research progress of human induced pluripotent stem cells in the establishment and application of dilated cardiomyopathy disease model . Acta Physiol Sin 2024; 76 (5): 775-782 (in Chinese with English abstract).