Gene editing and its applications in biomedicine

被引:0
作者
Guanglei Li
Xiangyang Li
Songkuan Zhuang
Liren Wang
Yifan Zhu
Yangcan Chen
Wen Sun
Zeguang Wu
Zhuo Zhou
Jia Chen
Xingxu Huang
Jin Wang
Dali Li
Wei Li
Haoyi Wang
Wensheng Wei
机构
[1] ShanghaiTech University,School of Life Science and Technology
[2] Shenzhen Second People’s Hospital,Department of Clinical Laboratory, Shenzhen Institute of Translational Medicine, The First Affiliated Hospital of Shenzhen University
[3] East China Normal University,Shanghai Frontiers Science Research Base of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences
[4] Chinese Academy of Sciences,State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology
[5] University of Chinese Academy of Sciences,Institute for Stem Cell and Regenerative Medicine
[6] Chinese Academy of Sciences,Biomedical Pioneering Innovation Center, Beijing Advanced Innovation Center for Genomics, Peking
[7] Peking University,Tsinghua Center for Life Sciences, Peking University Genome Editing Research Center, State Key Laboratory of Protein and Plant Gene Research, School of Life Sciences
[8] ShanghaiTech University,Gene Editing Center, School of Life Science and Technology
[9] Bejing Institute for Stem Cell and Regenerative Medicine,HIT Center for Life Sciences
[10] Harbin Institute of Technology,undefined
来源
Science China Life Sciences | 2022年 / 65卷
关键词
gene editing; CRISPR; high-throughput functional genomics; diagnostics; animal model; therapeutics;
D O I
暂无
中图分类号
学科分类号
摘要
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.
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收藏
页码:660 / 700
页数:40
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共 3599 条
  • [1] Abudayyeh OO(2016)C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector Science 353 aaf5573-284
  • [2] Gootenberg JS(2017)RNA targeting with CRISPR-Cas13 Nature 550 280-386
  • [3] Konermann S(2019)A cytosine deaminase for programmable single-base RNA editing Science 365 382-282
  • [4] Joung J(2020)Massively multiplexed nucleic acid detection with Cas13 Nature 582 277-1882
  • [5] Slaymaker IM(2016)A multiplexed single-cell CRISPR screening platform enables systematic dissection of the unfolded protein response Cell 167 1867-E9
  • [6] Cox DBT(2018)Large deletions induced by Cas9 cleavage Nature 560 E8-2413
  • [7] Shmakov S(2002)Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning Science 296 2410-458
  • [8] Makarova KS(2009)Gene therapy for immunodeficiency due to adenosine deaminase deficiency N Engl J Med 360 447-1364
  • [9] Semenova E(2010)Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms Mol Ther 18 1357-431
  • [10] Minakhin L(2020)Clades of huge phages from across Earth’s ecosystems Nature 578 425-573