描述基因组编辑的脱靶效应

IF 4.7 3区 工程技术 Q2 ENGINEERING, BIOMEDICAL Current Opinion in Biomedical Engineering Pub Date : 2023-06-17 DOI:10.1016/j.cobme.2023.100480
Xichen Rao , Huanan Zhao , Chuyun Shao , Chengqi Yi
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引用次数: 3

摘要

可编程的碱基编辑器和初始编辑器为高效和精确的基因组操作提供了潜力。然而,为了在临床环境中安全地应用这些编辑器,全面表征它们的脱靶效应是必不可少的。在这篇综述中,我们介绍了一些有代表性的技术来评估基因组和转录组的脱靶效应,并深入了解这些基因组编辑器的脱靶效应。重要的是,我们还讨论了目前体外和体内生物医学应用的脱靶评估方法的局限性,并强调需要开发全面和量身定制的脱靶评估方法,这对于推进治疗公司和研究小组推出的安全有效的基因编辑应用至关重要。
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Characterizing off-target effects of genome editors

Programmable base editors and prime editors offer the potential for highly efficient and precise genome manipulation. However, in order to safely apply these editors in clinical settings, it is essential to comprehensively characterize their off-target effects. In this review, we introduce some representative technologies for assessing off-target effects on both the genome and transcriptome and gain insight into the off-target effects of these genome editors. Importantly, we also discuss the limitations of the current off-target assessment methods for both ex vivo and in vivo biomedical applications and highlight the need to develop comprehensive and tailored off-target assessing methods which will be crucial for advancing the safe and effective gene editing applications launched by therapeutic companies and research groups.

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来源期刊
Current Opinion in Biomedical Engineering
Current Opinion in Biomedical Engineering Medicine-Medicine (miscellaneous)
CiteScore
8.60
自引率
2.60%
发文量
59
期刊最新文献
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