Inherited cardiac arrhythmias are a group of genetic diseases predisposing to sudden cardiac arrest, mainly resulting from variants in genes encoding cardiac ion channels or proteins involved in their regulation. Currently available therapeutic options (pharmacotherapy, ablative therapy and device-based therapy) can not preclude the occurrence of arrhythmia events and/or provide complete protection. With growing understanding of the genetic background and molecular mechanisms of inherited cardiac arrhythmias, advancing insight of stem cell technology, and development of vectors and delivery strategies, gene therapy and stem cell therapy may be promising approaches for treatment of inherited cardiac arrhythmias. Recent years have witnessed impressive progress in the basic science aspects and there is a clear and urgent need to be translated into the clinical management of arrhythmic events. In this review, we present a succinct overview of gene and cell therapy strategies, and summarize the current status of gene and cell therapy. Finally, we discuss future directions for implementation of gene and cell therapy in the therapy of inherited cardiac arrhythmias.

遗传性心律失常是一组容易导致心脏骤停的遗传性疾病，主要是由编码心脏离子通道的基因或参与其调节的蛋白质的变异引起的。目前可用的治疗选择（药物治疗、消融治疗和基于设备的治疗）不能排除心律失常事件的发生和/或提供完整的保护。随着对遗传性心律失常的遗传背景和分子机制的了解不断加深、干细胞技术的深入了解以及载体和递送策略的开发，基因治疗和干细胞治疗可能是治疗遗传性心律失常的有前途的方法。近年来，基础科学方面取得了令人瞩目的进展，显然迫切需要将其转化为心律失常事件的临床管理。在这篇综述中，我们对基因和细胞治疗策略进行了简要概述，并总结了基因和细胞治疗的现状。最后，我们讨论了遗传性心律失常治疗中基因和细胞疗法实施的未来方向。