CRISPR/Cas系统递送技术及其应用研究进展
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1.1)北京理工大学生命学院,前沿交叉科学研究院,医工融合研究院,分子医学与生物诊疗重点实验室,北京 100081;2.2)湖南中医药大学药学院,长沙 410208;3.3)郑州大学第一附属医院放疗科,郑州 450000

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基金项目:

国家自然科学基金(31901053, 31871003), 湖南省自然科学基金(2018JJ1019, 2019JJ50196)和湖湘青年英才项目(2018RS3094)联合资助.


Delivery and Application Progresses of CRISPR/Cas Gene Editing System
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Affiliation:

1.1)School of Life Science, Advanced Research Institute of Multidisciplinary Science, Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing 100081, China;2.2)School of Pharmacy, Hunan University of Chinese Medicine, Changsha 410208, China;3.3)Department of Radiation Oncology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou 450000, China

Fund Project:

This work was supported by grants from The National Natural Science Foundation of China (31901053, 31871003), the Hunan Provincial Natural Science Foundation of China (2018JJ1019, 2019JJ50196) and the Hu-Xiang Young Talent Program (2018RS3094).

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    摘要:

    基于CRISPR/Cas的基因编辑系统是近年来研究发展最重要的生物技术之一,其在基因编辑、核酸成像、转录调控、基因检测与疾病诊断、动物模型建立、农作物改良等领域均有十分广泛的应用. 本文主要介绍了CRISPR/Cas基因编辑技术的背景与发展历程,梳理了包括纳米载体在内的各类递送技术,总结了该技术应用于疾病治疗的临床前和临床研究进展,简述了CRISPR/Cas在其他更广泛领域的应用,并就该技术面临的挑战、发展趋势和应用前景做了展望.

    Abstract:

    CRISPR/Cas-based gene editing system is one of the newly-developed and promising biotechnologies. It shows broad application prospects for gene editing, DNA and RNA imaging, regulation of gene transcription, gene detection and disease diagnosis, establishing animal disease models, and improving crop, etc. In this review, we overviewed the background and development history of CRISPR / Cas systems, discussed recently-investigated delivery methods including various nanocarriers, summarize the preclinical and clinical advances of CRISPR/Cas-based therapeutics, and introduced other application areas besides gene editing. Finally, we made an outlook on the challenges we need pay attention to, and the future application prospects of CRISPR/Cas technologies.

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赵子璇,李春辉,周莉莉,赵德尧,翁郁华,夏新华,黄渊余. CRISPR/Cas系统递送技术及其应用研究进展[J].生物化学与生物物理进展,2020,47(4):286-299

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历史
  • 收稿日期:2019-11-20
  • 最后修改日期:2020-02-19
  • 接受日期:2020-03-10
  • 在线发布日期: 2020-08-11
  • 出版日期: 2020-04-20