Research progress and biotechnological applications of the prime editing

XU Zhimeng; XIE Zhen

doi:10.12211/2096-8280.2023-038

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Research progress and biotechnological applications of the prime editing

Invited Review | 更新时间：2025-03-20

- Research progress and biotechnological applications of the prime editing
- Synthetic Biology Journal Vol. 5, Issue 1, Pages: 1-15(2024)
- 作者机构：
  
  清华大学自动化系，北京信息科学与技术国家研究中心，生物信息学研究部，生物信息学教育部重点实验室，合成与系统生物学研究中心，北京 100084
- 作者简介：
- 基金信息：
- DOI：10.12211/2096-8280.2023-038
  CLC： Q812
- Received：08 June 2023，
  
  Revised：2023-09-08，
  
  Published：29 February 2024
- 稿件说明：
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许志锰，谢震. 引导编辑研究进展及其应用［J］. 合成生物学， 2024， 5（1）： 1-15

XU Zhimeng， XIE Zhen. Research progress and biotechnological applications of the prime editing［J］. Synthetic Biology Journal， 2024， 5（1）： 1-15
许志锰，谢震. 引导编辑研究进展及其应用［J］. 合成生物学， 2024， 5（1）： 1-15 DOI： 10.12211/2096-8280.2023-038.

XU Zhimeng， XIE Zhen. Research progress and biotechnological applications of the prime editing［J］. Synthetic Biology Journal， 2024， 5（1）： 1-15 DOI： 10.12211/2096-8280.2023-038.

摘要

引导编辑器（prime editor，PE）是继碱基编辑器（base editor，BE）之后新问世的基于CRISPR/Cas（clustered regularly interspaced short palindromic repeats/CRISPR-associated）系统的基因编辑工具，可以在不造成DNA双链断裂的情况下引入碱基替换、插入和删除。PE因其全面的编辑能力，问世即受到全球学者的广泛关注，然而PE表达盒编码较长（>6 kb）、编辑效率较低等问题也亟待研究人员解决。PE的研究方向与BE有许多相似之处，本文首先梳理了学界对PE本身编辑效率和安全性的探索；然后重点介绍了PE效应蛋白、pegRNA和其他细胞因子三个方面对PE的改进手段，以及为方便PE应用而开发的自动化设计工具；最后梳理了PE在动植物以及基因治疗中的应用。方兴未艾的PE领域尽管还难称完善，但在提高编辑效率和改进安全性等方面已取得了许多重要进展。鉴于Cas9、BE等基因编辑工具已广泛应用于遗传病疗法，PE走向遗传病治疗值得期待。

Abstract

Prime Editor (PE) is an innovative gene editing tool based on the Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein (CRISPR/Cas) system

which has revolutionized multiple fields

including genetics

medicine

and agriculture. Emerging as a successor to Base Editor (BE)

PE has gained worldwide attention due to its ability to introduce base substitutions

insertions

and deletions without causing double-strand DNA breaks

which significantly reduces the risk of off-target effect and unwanted genetic change. Notwithstanding its immense potential

researchers need to address PE's long encoding sequence and low editing efficiency for its maximal applications. Researchers have been working relentlessly to explore and enhance the editing efficiency and safety of PE by modifying its protein scaffold

optimizing the guide RNA design

and identifying cellular factors that influence its activity. Improved PE variants have been developed with enhanced accuracy and efficiency as well as decreased off-target effect when compared with their initial versions

demonstrating their potential in gene editing-related applications. Several strategies have been investigated to enhance PE performance

including: ① Modifying the structure of PE proteins to increase their efficiency

specificity

and binding affinity

thereby significantly improving their editing activity. ② Optimizing the design of pegRNAs

such as modifying the length

composition

or structure

that can boost PE's editing efficiency. ③ Identifying and manipulating cellular factors

such as proteins and RNAs

that bear functional relationships with the PE system

thus greatly enhancing its gene editing capabilities. ④ Developing automated design tools to facilitate the customization of the PE system for specific applications

vastly improving its practicality in research and clinical settings. Finally

this article summarizes the applications of PE in engineering animals and plants and developing gene therapy. Despite much room for further improvement in PE

significant advances have been made in improving its editing efficiency and safety. The rapid development of Cas9 and BE for treating genetic diseases stands as compelling testimony to the potential of PE in advancing gene editing technologies and applications. With continued research and development

PE holds great promise for improving human health and well-being.

关键词

Keywords

references

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JIN Fan

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Department of Automation， Tsinghua University， Tsinghua National Laboratory of Information Science and Technology， Center for Synthesis and Systems Biology， Key Laboratory of Bioinformatics， Ministry of Education， Department of Bioinformatics Research

Functional Nano and Soft Materials （FUNSOM）， Soochow University

Institute of Synthetic Biology， Shenzhen Institutes of Advanced Technology， Chinese Academy of Sciences

Department of Information Resources Management， School of Economics and Management， University of Chinese Academy of Sciences

National Science Library （Chengdu）， Chinese Academy of Sciences

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