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. 2022 Feb 18;65(4):660–700. doi: 10.1007/s11427-021-2057-0

Gene editing and its applications in biomedicine

Guanglei Li 1,#, Xiangyang Li 1,#, Songkuan Zhuang 2,#, Liren Wang 3,#, Yifan Zhu 3,#, Yangcan Chen 4,5,6,#, Wen Sun 4,6,#, Zeguang Wu 7,#, Zhuo Zhou 7,#, Jia Chen 8,✉, Xingxu Huang 1,✉, Jin Wang 2,✉, Dali Li 3,✉, Wei Li 4,5,6,9,10,✉, Haoyi Wang 4,5,6,✉, Wensheng Wei 7,✉
PMCID: PMC8889061  PMID: 35235150

Abstract

The steady progress in genome editing, especially genome editing based on the use of clustered regularly interspaced short palindromic repeats (CRISPR) and programmable nucleases to make precise modifications to genetic material, has provided enormous opportunities to advance biomedical research and promote human health. The application of these technologies in basic biomedical research has yielded significant advances in identifying and studying key molecular targets relevant to human diseases and their treatment. The clinical translation of genome editing techniques offers unprecedented biomedical engineering capabilities in the diagnosis, prevention, and treatment of disease or disability. Here, we provide a general summary of emerging biomedical applications of genome editing, including open challenges. We also summarize the tools of genome editing and the insights derived from their applications, hoping to accelerate new discoveries and therapies in biomedicine.

Keywords: gene editing, CRISPR, high-throughput functional genomics, diagnostics, animal model, therapeutics

Acknowledgements

This work was supported by the National Natural Science Foundation of China (81830004, 31922046, 31770057, 31722036, 31930016, 31870893), the Sanming Project of Medicine in Shenzhen (SZSM202011017), the National Key Research and Development Program of China (2018YFA0801401, 2019YFA0110800, 2018YFA0107703, 2019YFA0110000, 2020YFA0707800, 2020YFA0707600), the Strategic Priority Research Program of the Chinese Academy of Sciences (XDA16030403, XDA16010503), Beijing Municipal Science & Technology Commission (Z181100001318009), Beijing Advanced Innovation Center for Genomics at Peking University and the Peking-Tsinghua Center for Life Sciences, and the National Major Science & Technology Project for Control and Prevention of Major Infectious Diseases in China (2018ZX10301401). We are grateful to Dr. Gaofeng Fan from ShanghaiTech University for manuscript editing.

Footnotes

Compliance and ethics

The author(s) declare that they have no conflict of interest.

Contributed equally to this work

Contributor Information

Jia Chen, Email: chenjia@shanghaitech.edu.cn.

Xingxu Huang, Email: huangxx@shanghaitech.edu.cn.

Jin Wang, Email: wangj01@hotmail.com.

Dali Li, Email: dlli@bio.ecnu.edu.cn.

Wei Li, Email: liwei@ioz.ac.cn.

Haoyi Wang, Email: wanghaoyi@ioz.ac.cn.

Wensheng Wei, Email: wswei@pku.edu.cn.

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