The rapid invention of genome-editing technologies over the past decade, which has already been transformative for biomedical research, has raised the tantalizing prospect of an entirely new therapeutic modality. Whereas the treatment of chronic cardiovascular diseases has heretofore entailed the use of chronic therapies that typically must be taken repeatedly and frequently for the remainder of the lifetime, genome editing will enable the development of “one-and-done” therapies with durable effects. This Review summarizes the variety of available genome-editing approaches, including nuclease editing, base editing, epigenome editing, and prime editing; illustrates how these various approaches could be implemented as novel therapies for cardiovascular diseases; and outlines a path from technology development to preclinical studies to clinical trials. Although this Review focuses on PCSK9 as an instructive example of the various genome-editing approaches under active investigation, the lessons learned will be broadly applicable to the treatment of a variety of diseases.

中文翻译：

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转向心血管疾病的基因组编辑疗法

过去十年中基因组编辑技术的快速发明已经为生物医学研究带来了变革，为一种全新的治疗方式带来了诱人的前景。迄今为止，慢性心血管疾病的治疗需要使用通常必须在余生中反复且频繁地使用的慢性疗法，而基因组编辑将使开发具有持久效果的“一次性”疗法成为可能。本综述总结了各种可用的基因组编辑方法，包括核酸酶编辑、碱基编辑、表观基因组编辑和初始编辑；说明如何将这些不同的方法作为心血管疾病的新疗法实施；并概述了从技术开发到临床前研究再到临床试验的路径。虽然这篇评论的重点是PCSK9作为正在积极研究的各种基因组编辑方法的一个具有指导意义的例子，所吸取的经验教训将广泛适用于各种疾病的治疗。