【摘要】 儿茶酚胺敏感性多形性室速(catecholaminergic polymorphic ventricular tachycardia, CPVT)是一种由基因变异引起的致命性心律失常，具有极高的晕厥和猝死发生率，传统治疗方法效果有限。近年来，基因治疗作为一种潜在的治愈手段受到广泛关注，有望为CPVT患者提供更有效的治疗选择。本文综述了CPVT的基因特征以及基因治疗的最新研究进展，包括基因替代、突变等位基因抑制、CRISPR/Cas9基因编辑及生物通路抑制等策略，并总结了其临床应用前景与面临的挑战。更多还原

【Abstract】 Catecholamine-sensitive polymorphic ventricular tachycardia(CPVT) is a fatal cardiac arrhythmia caused by a genetic variant with a very high incidence of syncope and sudden cardiac death, and conventional treatments have limited effectiveness. In recent years, gene therapy has received widespread attention as a potential cure and is expected to provide more effective treatment options for CPVT patients. This article reviews the genetic features of CPVT and the latest research progress in gene therapy, including strategies such as gene replacement, mutant allele inhibition, CRISPR/Cas9 gene editing and biological pathway inhibition, and summarizes the prospects and challenges of its clinical application.更多还原