Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing.

Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing.
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DOI:
10.1038/s41467-023-35886-6
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发表时间:
2023-01-13
影响因子:
16.6
通讯作者:
Chiarle, Roberto
Chiarle, Roberto
中科院分区:
综合性期刊1区
文献类型:
--
作者:
Tao, Jianli;Bauer, Daniel E.;Chiarle, Roberto

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CRISPR-Cas基因编辑在过去的十年里彻底改变了实验分子生物学,并为人类遗传病的治疗带来了巨大的希望。本文回顾了CRISPR-Cas9/Cas12/Cas13核酸酶、DNA碱基编辑、主编辑和RNA碱基编辑的发展,重点评估和改进了它们的编辑精度和安全性,突破了编辑的特异性和效率的极限。我们总结了每个CRISPR工具从DNA编辑到RNA编辑的能力和局限性,并强调了未来改进和应用于基础研究的机会,以及在患者中使用它们的治疗和临床考虑。CRISPR-CAS工具在过去十年中在基因组工程方面显示出了非凡的前景。作者综述了CRISPR-Cas9/Cas12/Cas13核酸酶、DNA碱基编辑、基序编辑和RNA碱基编辑的发展,以及它们的编辑精度、非靶点效应和临床考虑。
CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past decade and holds great promise for the treatment of human genetic diseases. Here we review the development of CRISPR-Cas9/Cas12/Cas13 nucleases, DNA base editors, prime editors, and RNA base editors, focusing on the assessment and improvement of their editing precision and safety, pushing the limit of editing specificity and efficiency. We summarize the capabilities and limitations of each CRISPR tool from DNA editing to RNA editing, and highlight the opportunities for future improvements and applications in basic research, as well as the therapeutic and clinical considerations for their use in patients. CRISPR-Cas tools have shown exceptional promise in genome engineering over the past decade. Here the authors review the development of CRISPR-Cas9/Cas12/Cas13 nucleases, DNA base editors, prime editors, and RNA base editors, as well as their editing precision, off-target effects, and clinical considerations.
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