Kamdar Forum, Klaassen Kamdar Andre, Koyano-Nakagawa Naoko, Garry Mary G, Garry Daniel J
Cardiovascular Division and Lillehei Heart Institute, University of Minnesota, Minneapolis, Minnesota.
Cardiovascular Division and Lillehei Heart Institute, University of Minnesota, Minneapolis, Minnesota.
J Card Fail. 2015 Sep;21(9):761-70. doi: 10.1016/j.cardfail.2015.04.010. Epub 2015 Apr 28.
Inherited cardiomyopathies, including hypertrophic cardiomyopathy, dilated cardiomyopathies, arrythmogenic right ventricular cardiomyopathy, and other inherited forms of heart failure, represent a unique set of genetically defined cardiovascular disease processes. Unraveling the molecular mechanisms of these deadly forms of human heart disease has been challenging, but recent groundbreaking scientific advances in stem cell technology have allowed for the generation of patient-specific human inducible stem cell (hiPSC)-derived cardiomyocytes (CMs). hiPSC-derived CMs retain the genetic blueprint of the patient, they can be maintained in culture, and they recapitulate the phenotypic characteristics of the disease in vitro, thus serving as a disease in a dish. This review provides an overview of in vitro modeling of inherited cardiomyopathies with the use of patient-specific hiPSC-derived CMs.
遗传性心肌病，包括肥厚型心肌病、扩张型心肌病、致心律失常性右室心肌病以及其他遗传性心力衰竭形式，代表了一组独特的由基因定义的心血管疾病进程。阐明这些致命形式的人类心脏病的分子机制一直具有挑战性，但干细胞技术最近取得的突破性科学进展使得能够生成患者特异性的人诱导多能干细胞（hiPSC）衍生的心肌细胞（CMs）。hiPSC衍生的CMs保留了患者的基因蓝图，可以在培养中维持，并且在体外重现疾病的表型特征，从而成为培养皿中的疾病。本综述概述了利用患者特异性hiPSC衍生的CMs对遗传性心肌病进行体外建模的情况。
