【摘要】 心力衰竭是心肌结构和功能变化导致心脏舒缩功能障碍的临床综合征。终末期心功能不全是心力衰竭最严重的阶段，表现为心脏泵血能力严重衰退，即使接受药物、手术和器械治疗，仍持续出现严重心衰症状。随着心力衰竭分子机制探索的深入，药物对许多新型治疗靶点与信号通路的调控存在局限性，基因疗法已成为干预终末期心功能不全的重要治疗手段，基因治疗可以将外源正常基因导入细胞，靶向修复导致心肌损伤的基因缺陷，进而延缓终末期心功能不全的发生。近年来，基因治疗在终末期心功能不全的基础和临床研究方面取得了一定进展。本文将综述终末期心功能不全基因治疗及其递送系统的最新进展，以期为寻找终末期心功能不全的潜在基因治疗靶点提供参考。更多还原

【Abstract】 Heart failure is a clinical syndrome caused by changes in myocardial structure and function leading to impaired cardiac systolic and diastolic function.End-stage heart failure is the most severe stage of heart failure, characterized by a severe decline in the heart’s pumping capacity, with persistent severe heart failure symptoms even after drug, surgical, and device-assisted treatment.With the deepening exploration of the molecular mechanisms of heart failure, drugs have limitations in regulating many novel therapeutic targets and signaling pathways, gene therapy has become an important treatment for end-stage heart failure.Gene therapy can introduce exogenous normal genes into cells to target and repair gene defects that cause myocardial damage, thereby delaying the onset of end-stage heart failure.In recent years, gene therapy has made significant discoveries in basic and clinical research on end-stage heart failure.This article reviews the latest advances in gene therapy and delivery systems for end-stage heart failure, aiming to provide a reference for identifying potential gene therapy targets for end-stage heart failure.更多还原