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Base editors: development and applications in biomedicine

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Abstract

Base editor (BE) is a gene-editing tool developed by combining the CRISPR/Cas system with an individual deaminase, enabling precise single-base substitution in DNA or RNA without generating a DNA double-strand break (DSB) or requiring donor DNA templates in living cells. Base editors offer more precise and secure genome-editing effects than other conventional artificial nuclease systems, such as CRISPR/Cas9, as the DSB induced by Cas9 will cause severe damage to the genome. Thus, base editors have important applications in the field of biomedicine, including gene function investigation, directed protein evolution, genetic lineage tracing, disease modeling, and gene therapy. Since the development of the two main base editors, cytosine base editors (CBEs) and adenine base editors (ABEs), scientists have developed more than 100 optimized base editors with improved editing efficiency, precision, specificity, targeting scope, and capacity to be delivered in vivo, greatly enhancing their application potential in biomedicine. Here, we review the recent development of base editors, summarize their applications in the biomedical field, and discuss future perspectives and challenges for therapeutic applications.

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Acknowledgements

This work was financially supported by National Natural Science Foundation of China (No. 32100410); National Key Research and Development Program of China (No. 2022YFA1105403); Science and Technology Program of Guangzhou (Nos. 202201010409 and 2023A04J0732); Major Science and Technology Project of Hainan Province (No. ZDKJ2021030); 2020 Research Program of Sanya Yazhou Bay Science and Technology City (No. 202002011); Science and Technology Planning Project of Guangdong Province, China (Nos. 2020B1212060052 and 2021B1212040016); Research Unit of Generation of Large Animal Disease Models, Chinese Academy of Medical Sciences (No. 2019-I2M-5-025).

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Author notes

  1. These authors contributed equally to this work.

Authors and Affiliations

  1. China-New Zealand Joint Laboratory on Biomedicine and Health, CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Centre for Regenerative Medicine and Health, Hong Kong Institute of Science and Innovation, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, 510530, China

    Yanhui Liang, Fangbing Chen, Kepin Wang & Liangxue Lai

  2. Research Unit of Generation of Large Animal Disease Models, Chinese Academy of Medical Sciences (2019RU015), Guangzhou, 510530, China

    Yanhui Liang, Fangbing Chen, Kepin Wang & Liangxue Lai

  3. Sanya Institute of Swine Resource, Hainan Provincial Research Centre of Laboratory Animals, Sanya, 572000, China

    Yanhui Liang, Fangbing Chen, Kepin Wang & Liangxue Lai

  4. Guangdong Provincial Key Laboratory of Large Animal Models for Biomedicine, Wuyi University, Jiangmen, 529020, China

    Fangbing Chen, Kepin Wang & Liangxue Lai

Authors
  1. Yanhui Liang
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  2. Fangbing Chen
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  3. Kepin Wang
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  4. Liangxue Lai
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Corresponding author

Correspondence to Liangxue Lai.

Ethics declarations

Yanhui Liang, Fangbing Chen, Kepin Wang, and Liangxue Lai declare that they have no conflict of interest. This manuscript is a review article and does not involve a research protocol requiring approval by the relevant institutional review board or ethics committee.

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Liang, Y., Chen, F., Wang, K. et al. Base editors: development and applications in biomedicine. Front. Med. 17, 359–387 (2023). https://doi.org/10.1007/s11684-023-1013-y

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  • DOI: https://doi.org/10.1007/s11684-023-1013-y

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