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 Chen⁸, Xingxu Huang⁹, Jin Wang¹⁰, Dali Li¹¹, Wei Li^{12

13

14

15

16}, Haoyi Wang^{17

18

19}, Wensheng Wei²⁰

Affiliations

¹ School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China.
² Department of Clinical Laboratory, Shenzhen Institute of Translational Medicine, The First Affiliated Hospital of Shenzhen University, Shenzhen Second People's Hospital, Shenzhen, 518035, China.
³ Shanghai Frontiers Science Research Base of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.
⁴ State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China.
⁵ University of Chinese Academy of Sciences, Beijing, 100049, China.
⁶ Institute for Stem Cell and Regenerative Medicine, Chinese Academy of Sciences, Beijing, 100101, China.
⁷ Biomedical Pioneering Innovation Center, Beijing Advanced Innovation Center for Genomics, Peking-Tsinghua Center for Life Sciences, Peking University Genome Editing Research Center, State Key Laboratory of Protein and Plant Gene Research, School of Life Sciences, Peking University, Beijing, 100871, China.
⁸ Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China. chenjia@shanghaitech.edu.cn.
⁹ School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China. huangxx@shanghaitech.edu.cn.
¹⁰ Department of Clinical Laboratory, Shenzhen Institute of Translational Medicine, The First Affiliated Hospital of Shenzhen University, Shenzhen Second People's Hospital, Shenzhen, 518035, China. wangj01@hotmail.com.
¹¹ Shanghai Frontiers Science Research Base of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China. dlli@bio.ecnu.edu.cn.
¹² State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China. liwei@ioz.ac.cn.
¹³ University of Chinese Academy of Sciences, Beijing, 100049, China. liwei@ioz.ac.cn.
¹⁴ Institute for Stem Cell and Regenerative Medicine, Chinese Academy of Sciences, Beijing, 100101, China. liwei@ioz.ac.cn.
¹⁵ Bejing Institute for Stem Cell and Regenerative Medicine, Beijing, 100101, China. liwei@ioz.ac.cn.
¹⁶ HIT Center for Life Sciences, Harbin Institute of Technology, Harbin, 150001, China. liwei@ioz.ac.cn.
¹⁷ State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China. wanghaoyi@ioz.ac.cn.
¹⁸ University of Chinese Academy of Sciences, Beijing, 100049, China. wanghaoyi@ioz.ac.cn.
¹⁹ Institute for Stem Cell and Regenerative Medicine, Chinese Academy of Sciences, Beijing, 100101, China. wanghaoyi@ioz.ac.cn.
²⁰ Biomedical Pioneering Innovation Center, Beijing Advanced Innovation Center for Genomics, Peking-Tsinghua Center for Life Sciences, Peking University Genome Editing Research Center, State Key Laboratory of Protein and Plant Gene Research, School of Life Sciences, Peking University, Beijing, 100871, China. wswei@pku.edu.cn.

^# Contributed equally.

PMID: 35235150
PMCID: PMC8889061
DOI: 10.1007/s11427-021-2057-0

Review

Gene editing and its applications in biomedicine

Guanglei Li et al. Sci China Life Sci. 2022 Apr.

. 2022 Apr;65(4):660-700.

doi: 10.1007/s11427-021-2057-0. Epub 2022 Feb 18.

Authors

Affiliations

¹ School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China.
² Department of Clinical Laboratory, Shenzhen Institute of Translational Medicine, The First Affiliated Hospital of Shenzhen University, Shenzhen Second People's Hospital, Shenzhen, 518035, China.
³ Shanghai Frontiers Science Research Base of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.
⁴ State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China.
⁵ University of Chinese Academy of Sciences, Beijing, 100049, China.
⁶ Institute for Stem Cell and Regenerative Medicine, Chinese Academy of Sciences, Beijing, 100101, China.
⁷ Biomedical Pioneering Innovation Center, Beijing Advanced Innovation Center for Genomics, Peking-Tsinghua Center for Life Sciences, Peking University Genome Editing Research Center, State Key Laboratory of Protein and Plant Gene Research, School of Life Sciences, Peking University, Beijing, 100871, China.
⁸ Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China. chenjia@shanghaitech.edu.cn.
⁹ School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China. huangxx@shanghaitech.edu.cn.
¹⁰ Department of Clinical Laboratory, Shenzhen Institute of Translational Medicine, The First Affiliated Hospital of Shenzhen University, Shenzhen Second People's Hospital, Shenzhen, 518035, China. wangj01@hotmail.com.
¹¹ Shanghai Frontiers Science Research Base of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China. dlli@bio.ecnu.edu.cn.
¹² State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China. liwei@ioz.ac.cn.
¹³ University of Chinese Academy of Sciences, Beijing, 100049, China. liwei@ioz.ac.cn.
¹⁴ Institute for Stem Cell and Regenerative Medicine, Chinese Academy of Sciences, Beijing, 100101, China. liwei@ioz.ac.cn.
¹⁵ Bejing Institute for Stem Cell and Regenerative Medicine, Beijing, 100101, China. liwei@ioz.ac.cn.
¹⁶ HIT Center for Life Sciences, Harbin Institute of Technology, Harbin, 150001, China. liwei@ioz.ac.cn.
¹⁷ State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China. wanghaoyi@ioz.ac.cn.
¹⁸ University of Chinese Academy of Sciences, Beijing, 100049, China. wanghaoyi@ioz.ac.cn.
¹⁹ Institute for Stem Cell and Regenerative Medicine, Chinese Academy of Sciences, Beijing, 100101, China. wanghaoyi@ioz.ac.cn.
²⁰ Biomedical Pioneering Innovation Center, Beijing Advanced Innovation Center for Genomics, Peking-Tsinghua Center for Life Sciences, Peking University Genome Editing Research Center, State Key Laboratory of Protein and Plant Gene Research, School of Life Sciences, Peking University, Beijing, 100871, China. wswei@pku.edu.cn.

^# Contributed equally.

PMID: 35235150
PMCID: PMC8889061
DOI: 10.1007/s11427-021-2057-0

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: CRISPR; animal model; diagnostics; gene editing; high-throughput functional genomics; therapeutics.

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References

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Gene editing and its applications in biomedicine

Affiliations

Gene editing and its applications in biomedicine

Authors

Affiliations

Abstract

References

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