Advancing CRISPR base editing technology through innovative strategies and ideas.

IF 8 2区 生物学 Q1 BIOLOGY Science China Life Sciences Pub Date : 2024-09-02 DOI:10.1007/s11427-024-2699-5
Xiongwei Fan, Yang Lei, Liren Wang, Xiushan Wu, Dali Li
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Abstract

The innovation of CRISPR/Cas gene editing technology has developed rapidly in recent years. It is widely used in the fields of disease animal model construction, biological breeding, disease diagnosis and screening, gene therapy, cell localization, cell lineage tracking, synthetic biology, information storage, etc. However, developing idealized editors in various fields is still a goal for future development. This article focuses on the development and innovation of non-DSB editors BE and PE in the platform-based CRISPR system. It first explains the application of ideas for improvement such as "substitution", "combination", "adaptation", and "adjustment" in BE and PE development and then catalogues the ingenious inversions and leaps of thought reflected in the innovations made to CRISPR technology. It will then elaborate on the efforts currently being made to develop small editors to solve the problem of AAV overload and summarize the current application status of editors for in vivo gene modification using AAV as a delivery system. Finally, it summarizes the inspiration brought by CRISPR/Cas innovation and assesses future prospects for development of an idealized editor.

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通过创新战略和理念推进 CRISPR 碱基编辑技术。
近年来,CRISPR/Cas 基因编辑技术创新发展迅速。它被广泛应用于疾病动物模型构建、生物育种、疾病诊断与筛选、基因治疗、细胞定位、细胞系追踪、合成生物学、信息存储等领域。然而,开发各领域的理想化编辑器仍是未来发展的目标。本文主要介绍非DSB编辑器BE和PE在基于平台的CRISPR系统中的发展与创新。文章首先解释了 "替代"、"组合"、"适应 "和 "调整 "等改良思想在 BE 和 PE 开发中的应用,然后列举了 CRISPR 技术创新中体现出的巧妙反转和思维飞跃。然后阐述目前为解决 AAV 过载问题而开发小型编辑器的努力,并总结编辑器在以 AAV 为传递系统进行体内基因修饰方面的应用现状。最后,它将总结 CRISPR/Cas 创新带来的启示,并评估理想化编辑器的未来发展前景。
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来源期刊
CiteScore
15.10
自引率
8.80%
发文量
2907
审稿时长
3.2 months
期刊介绍: Science China Life Sciences is a scholarly journal co-sponsored by the Chinese Academy of Sciences and the National Natural Science Foundation of China, and it is published by Science China Press. The journal is dedicated to publishing high-quality, original research findings in both basic and applied life science research.
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