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.
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 recentlyinvestigated 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.
作者
赵子璇
李春辉
周莉莉
赵德尧
翁郁华
夏新华
黄渊余
ZHAO Zi-Xuan;LI Chun-Hui;ZHOU Li-Li;ZHAO De-Yao;WENG Yu-Hua;XIA Xin-Hua;HUANG Yuan-Yu(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;School of Pharmacy,Hunan University of Chinese Medicine,Changsha 410208,China;Department of Radiation Oncology,The First Affiliated Hospital of Zhengzhou University,Zhengzhou 450000,China)
出处
《生物化学与生物物理进展》
SCIE
CAS
CSCD
北大核心
2020年第4期286-299,共14页
Progress In Biochemistry and Biophysics
基金
国家自然科学基金(31901053,31871003)
湖南省自然科学基金(2018JJ1019,2019JJ50196)
湖湘青年英才项目(2018RS3094)联合资助.