Gene therapy has recently become a reality in the treatment of cardiovascular diseases. Strategies to modulate gene expression using antisense oligonucleotides or small interfering RNA are proving to be safe and effective in the clinic. Adeno‐associated viral vector‐based gene delivery and CRISPR‐Cas9‐based genome editing have emerged as efficient strategies for gene delivery and repair in humans. Overall, gene therapy holds the promise not only of expanding current treatment options, but also of intervening in previously untackled causal disease mechanisms with little side effects. This scientific statement provides a comprehensive overview of the various modalities of gene therapy used to treat heart failure and some of its risk factors, and their application in the clinical setting. It discusses specifically the possibilities of gene therapy for hereditary heart diseases and (non)‐genetic heart failure. Furthermore, it addresses safety and clinical trial design issues and challenges for future regulatory strategies.

中文翻译：

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心力衰竭基因治疗的最新进展和前景。ESC心力衰竭协会、ESC心血管基因组学委员会以及ESC心肌和心包疾病工作组的科学声明


基因治疗最近已成为心血管疾病治疗领域的现实。使用反义寡核苷酸或小干扰 RNA 调节基因表达的策略在临床上被证明是安全有效的。基于腺相关病毒载体的基因递送和基于 CRISPR-Cas9 的基因组编辑已成为人类基因递送和修复的有效策略。总体而言，基因治疗不仅有望扩大当前的治疗选择，而且还有望干预以前未解决的致病机制，而且副作用很小。本科学声明全面概述了用于治疗心力衰竭的各种基因治疗方式及其一些风险因素，以及它们在临床环境中的应用。它特别讨论了基因治疗遗传性心脏病和（非）遗传性心力衰竭的可能性。此外，它还解决了安全和临床试验设计问题以及未来监管策略的挑战。