新一代基因编辑工具研究进展

王瑞; 周欣洁; 杜熙钦; 郝翟; 王琛; 邹秉杰; 宋沁馨; 周国华

doi:10.11665/j.issn.1000-5048.20220601

新一代基因编辑工具研究进展

1.
中国药科大学药物质量与安全预警教育部重点实验室，南京 210009
2.
南京大学生命分析化学国家重点实验室，南京 210093
3.
东部战区总医院临床药学科，南京 210002

基金项目: 国家自然科学基金资助项目（No.82173780）；药物质量与安全预警教育部重点实验室资助项目（No.DQCP20/21MS04）；国家级大学生创新创业训练计划项目成果（No.202210316001Z，No.202210316009Z）

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出版历程
- 收稿日期: 2022-12-03
- 修回日期: 2022-12-19
- 刊出日期: 2022-12-24

Research progress of next-generation gene editing tools

1.
Key Laboratory of Drug Quality Control and Pharmacovigilance of the Ministry of Education, China Pharmaceutical University, Nanjing 210009
2.
Department of Chemistry,State Key Laboratory of Analytical Chemistry for Life Science,Nanjing University, Nanjing 210093
3.
Department of Clinical Pharmacy, Jinling Hospital, Medical School of Nanjing University, Nanjing 210002, China

Funds: This study was funded by the National Natural Science Foundation of China (No.82173780), the Open Project Program of MOE Key Laboratory of Drug Quality Control and Pharmacovigilance (No.DQCP20/21MS04); National Innovation and Entrepreneurship Training Program for Undergraduate (No.202210316001Z and 202210316009Z)

摘要

摘要: 目前，以核酸酶为主要成分的基因编辑工具已经成功实现可编程地对哺乳动物基因组进行靶向突变或插入删除，从锌指核酸酶（ZFNs）、转录激活子样效应子核酸酶（TALENs）、CRISPR/Cas系统发展到更安全更精准的Cas9融合蛋白基因编辑工具和其他核酸酶基因编辑工具，本文系统阐述了基因编辑的发展演进历程、介绍了新一代基因编辑工具的开发与优化，针对基因编辑工具的临床应用与面临的挑战进行了展望。
- 基因编辑 /
- CRISPR/Cas9 /
- 碱基编辑 /
- 先导编辑 /
- DNA双链断裂 /
- 进展
Abstract: Gene editing tools with nucleases as the main component have now implemented programmable targeted mutagenesis or insertion or deletion of mammalian genomes.From zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), CRISPR/Cas system to safer and more accurate Cas9 fusion protein gene editing tools and other nuclease gene editing tools, this paper systematically describes the development and evolution of gene editing, with detailed introduction to the development and optimization of next-generation gene editing tools, and a prospect of the clinical application of and challenges for gene editing tools.
- gene editing /
- CRISPR/Cas9 /
- base editing /
- prime editing /
- double-strand break /
- progress

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