For a long time, gene editing had been a scientific concept, which was limited to a few applications. With recent developments, following the discovery of TALEN zinc-finger endonucleases and in particular the CRISPR/Cas system, gene editing has become a technique applicable in most laboratories. The current gain- and loss-of function models in basic science are revolutionary as they allow unbiased screens of unprecedented depth and complexity and rapid development of transgenic animals. Modifications of CRISPR/Cas have been developed to precisely interrogate epigenetic regulation or to visualize DNA complexes. Moreover, gene editing as a clinical treatment option is rapidly developing with first trials on the way. This article reviews the most recent progress in the field, covering expert opinions gathered during joint conferences on genome editing of the German Cardiac Society (DGK) and the German Center for Cardiovascular Research (DZHK). Particularly focusing on the translational aspect and the combination of cellular and animal applications, the authors aim to provide direction for the development of the field and the most frequent applications with their problems.

长期以来，基因编辑一直是一个科学概念，仅限于少数应用。随着最近的发展，随着 TALEN 锌指核酸内切酶的发现，特别是 CRISPR/Cas 系统的发现，基因编辑已成为大多数实验室适用的技术。当前基础科学中的功能增益和损失模型是革命性的，因为它们允许对转基因动物进行前所未有的深度和复杂性的无偏筛选和快速发展。已经开发了 CRISPR/Cas 的修饰来精确询问表观遗传调控或可视化 DNA 复合物。此外，基因编辑作为一种临床治疗选择正在迅速发展，初步试验正在进行中。本文回顾了该领域的最新进展，涵盖在德国心脏病学会 (DGK) 和德国心血管研究中心 (DZHK) 基因组编辑联合会议期间收集的专家意见。作者特别关注转化方面以及细胞和动物应用的结合，旨在为该领域的发展和最常见的应用提供方向。