诱导性多能干细胞联合基因编辑技术在遗传性心脏病研究中的应用进展

Combined Application of Induced Pluripotent Stem Cells and Gene Editing Technology in the Research of Inherited Heart Disease

摘要: 遗传性心脏病是发生恶性心律失常和心源性猝死的主要原因，但目前对其发生机制的研究还处于起步阶段。基因编辑技术的发展使遗传性心脏病的根治成为可能。综述遗传性心脏病的分子机制、诱导性多能干细胞（induced pluripotent stem cell,iPSC）及其分化的心肌细胞（iPSC-derived cardiomyocyte,iPSC-CM）与基因编辑技术的研究进展，重点介绍iPSC，iPSC-CM与基因编辑技术在建立遗传性心脏病的人源性细胞模型和探索治疗新方式方面的应用。

Abstract: Inherited heart disease is the main cause of malignant arrhythmia and sudden cardiac death, but little is known about its mechanism. Advances in gene editing technology have made it possible to cure inherited heart disease. Research progress in the molecular mechanism of inherited heart disease, induced pluripotent stem cell (iPSC), iPSC-derived cardiomyocyte (iPSC-CM) and gene editing technology were reviewed, with focus on the application of iPSC, iPSC-CM and gene editing technology in establishing human cell models of inherited heart disease and exploring new treatment options.