使用基础编辑器进行精确的基因组编辑。

Medical review (Berlin, Germany) Pub Date : 2023-02-22 eCollection Date: 2023-02-01 DOI:10.1515/mr-2022-0044
Hongcai Liu, Yao Zhu, Minjie Li, Zhimin Gu
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引用次数: 1

摘要

单核苷酸变异约占人类已知致病基因变异的一半。通过以最小副作用逆转致病点突变的基因组编辑策略具有巨大的治疗潜力,目前正在积极探索。精确有效的基因组编辑策略(如碱基编辑和引物编辑)的出现为核苷酸转换提供了强大的工具,而不会诱导双链DNA断裂(DSBs),这在治疗遗传疾病方面显示出巨大的潜力。开发了一个多样化的基础编辑器工具包,以提高不同应用环境下的编辑效率和准确性。在这里,我们总结了基础编辑(BEs)的发展、它们的局限性以及基于基础编辑的治疗策略的未来前景。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Precise genome editing with base editors.

Single-nucleotide variants account for about half of known pathogenic genetic variants in human. Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively pursued. The emerge of precise and efficient genome editing strategies such as base editing and prime editing provide powerful tools for nucleotide conversion without inducing double-stranded DNA breaks (DSBs), which have shown great potential for curing genetic disorders. A diverse toolkit of base editors has been developed to improve the editing efficiency and accuracy in different context of application. Here, we summarized the evolving of base editors (BEs), their limitations and future perspective of base editing-based therapeutic strategies.

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