Peking-Tsinghua Center for Life Sciences, Peking University, Beijing100871, China.
Academy for Advanced Interdisciplinary Studies, Peking University, Beijing100871, China.
ACS Chem Biol. 2023 Feb 17;18(2):205-217. doi: 10.1021/acschembio.2c00836. Epub 2023 Feb 2.
Various genome editing tools have been developed for programmable genome manipulation at specified genomic loci. However, it is crucial to comprehensively interrogate the off-target effect induced by these genome editing tools, especially when apply them onto the therapeutic applications. Here, we outlined the off-target effect that has been observed for various genome editing tools. We also reviewed detection methods to determine or evaluate the off-target editing, and we have discussed their advantages and limitations. Additionally, we have summarized current RNA editing tools for RNA therapy and medicine that may serve as alternative approaches for genome editing tools in both research and clinical applications.
各种基因组编辑工具已被开发用于在特定基因组位点进行可编程的基因组操作。然而,全面研究这些基因组编辑工具所诱导的脱靶效应至关重要,尤其是在将它们应用于治疗应用时。在这里,我们概述了各种基因组编辑工具所观察到的脱靶效应。我们还回顾了用于确定或评估脱靶编辑的检测方法,并讨论了它们的优点和局限性。此外,我们总结了当前用于 RNA 治疗和医学的 RNA 编辑工具,它们可能在研究和临床应用中作为基因组编辑工具的替代方法。