hiPSC Modeling of Inherited Cardiomyopathies.
hiPSC Modeling of Inherited Cardiomyopathies.
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DOI：
10.1007/s11936-014-0320-7
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发表时间：
2014-07
影响因子：
--
通讯作者：
Bernstein, Daniel
中科院分区：
文献类型：
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作者：
Jung, Gwanghyun;Bernstein, Daniel
人诱导多能干细胞衍生的心肌细胞（hiPSC-CMs）代表了研究遗传性心肌病的基本机制的强大的新模型系统。hiPSC-CM已被用于模拟几种心血管疾病，在遗传性心律失常方面取得了最大的成功，包括长QT和Timothy综合征以及致心律失常性右心室发育不良（ARVD）。最近，研究已经将hiPSC-CM应用于扩张型（DCM）和肥厚型（HCM）心肌病的研究，为疾病的基本机制提供了新的见解。然而，hiPSC-CM并不重现成熟人心肌细胞的许多结构和功能方面，而是反映了不成熟的胚胎或胎儿表型。因此，还有许多工作要做，以更好地理解这些差异，以及开发方法，以诱导hiPSC-CM成为一个完全成熟的表型。尽管存在这些局限性，hiPSC-CM代表了目前人类心脏的最佳体外相关性，并且是寻找心肌病潜在机制和筛选新药物治疗的宝贵工具。
Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) represent a powerful new model system to study the basic mechanisms of inherited cardiomyopathies. hiPSC-CMs have been utilized to model several cardiovascular diseases, achieving the most success in the inherited arrhythmias, including long QT and Timothy syndromes and arrhythmogenic right ventricular dysplasia (ARVD). Recently, studies have applied hiPSC-CMs to the study of both dilated (DCM) and hypertrophic (HCM) cardiomyopathies, providing new insights into basic mechanisms of disease. However, hiPSC-CMs do not recapitulate many of the structural and functional aspects of mature human cardiomyocytes, instead mirroring an immature, embryonic or fetal, phenotype. Thus, much work remains to better understand these differences as well as to develop methods to induce hiPSC-CMs into a fully mature phenotype. Despite these limitations, hiPSC-CMs represent the best current in vitro correlate of the human heart and an invaluable tool in the search for mechanisms underlying cardiomyopathy and for screening new pharmacologic therapies.