Department of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston Texas, USA.
Curr Opin Cardiol. 2020 May;35(3):242-248. doi: 10.1097/HCO.0000000000000723.
This review highlights recent progress in applying genome editing to the study and treatment of cardiovascular disease (CVD).
Recent work has shown that genome editing can be used to determine the pathogenicity of variants of unknown significance in patient-derived induced pluripotent stem cells. These cells can also be used to test therapeutic genome editing approaches in a personalized manner. Somatic genome editing holds great promise for the treatment of CVD, and important proof of concept experiments have already been performed in animal models. Here we briefly review recent progress in patient-derived cells, as well as the development of somatic genome-editing therapies for CVD, with a particular focus on liver and heart.
Translating this technology into the clinic will require precise editing enzymes, efficient delivery systems, and mitigation of off-target events and immune responses. Further development of these technologies will improve diagnostics and enable permanent correction of some of the most severe forms of CVD.
本文重点介绍了基因组编辑在心血管疾病（CVD）研究和治疗中的最新进展。
最近的研究表明，基因组编辑可用于确定患者来源的诱导多能干细胞中未知意义变异的致病性。这些细胞还可以用于以个性化的方式测试治疗性基因组编辑方法。体细胞基因组编辑为 CVD 的治疗提供了巨大的希望，并且已经在动物模型中进行了重要的概念验证实验。在这里，我们简要回顾了患者来源细胞的最新进展，以及针对 CVD 的体细胞基因组编辑疗法的发展，特别关注肝脏和心脏。
将这项技术转化为临床应用需要精确的编辑酶、高效的递送系统，以及减轻脱靶效应和免疫反应。这些技术的进一步发展将改善诊断，并能够永久性纠正一些最严重的 CVD 形式。
