Current advances in overcoming obstacles of CRISPR/Cas9 off-target genome editing

被引：21
作者：
Aquino-Jarquin, Guillermo [1 ,2 ]
机构：
[1] Hosp Infantil Mexico Dr Federico Gomez, Lab Invest Genom Genet & Bioinformat, Dr Marquez 162, Ciudad De Mexico 06720, Mexico
[2] Univ Autonoma Metropolitana, Dept Ciencias Nat, Unidad Cuajimalpa, Ciudad De Mexico, Mexico
来源：
MOLECULAR GENETICS AND METABOLISM | 2021年 / 134卷 / 1-2期
关键词：
CRISPR; Cas9; Genome-editing; Off-target effects; Base editors; Prime editing; Anti-CRISPR proteins; DOUBLE-STRANDED BREAKS; DNA CLEAVAGE; CAS9; BASE; CRISPR-CAS9; SPECIFICITY; NUCLEASES; CHALLENGES; SYSTEMS; DAMAGE;
D O I：
10.1016/j.ymgme.2021.08.002
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
CRISPR/Cas9-based technology has revolutionized biomedical research by providing a high-fidelity gene-editing method, foreshadowing a significant impact on the therapeutics of many human genetic disorders previously considered untreatable. However, off-target events represent a critical hurdle before genome editing can be fully established in clinical practice. This mini-review recapitulates some recent advances for detecting and overcoming off-target effects mediated by the CRISPR/Cas9 system that could increase the likelihood of clinical success of the CRISPR-based approaches. (c) 2021 Elsevier Inc. All rights reserved.
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收藏
页码：77 / 86
页数：10
相关论文
共 114 条
[1]   Repair of U/G and U/A in DNA by UNG2-associated repair complexes takes place predominantly by short-patch repair both in proliferating and growth-arrested cells [J].
Akbari, M ;
Otterlei, M ;
Peña-Diaz, J ;
Aas, PA ;
Kavli, B ;
Liabakk, NB ;
Hagen, L ;
Imai, K ;
Durandy, A ;
Slupphaug, G ;
Krokan, HE .
NUCLEIC ACIDS RESEARCH, 2004, 32 (18) :5486-5498
[2]   In vivo CRISPR editing with no detectable genome-wide off-target mutations [J].
Akcakaya, Pinar ;
Bobbin, Maggie L. ;
Guo, Jimmy A. ;
Malagon-Lopez, Jose ;
Clement, Kendell ;
Garcia, Sara P. ;
Fellows, Mick D. ;
Porritt, Michelle J. ;
Firth, Mike A. ;
Carreras, Alba ;
Baccega, Tania ;
Seeliger, Frank ;
Bjursell, Mikael ;
Tsai, Shengdar Q. ;
Nguyen, Nhu T. ;
Nitsch, Roberto ;
Mayr, Lorenz M. ;
Pinello, Luca ;
Bohlool-Y, Mohammad ;
Aryee, Martin J. ;
Maresca, Marcello ;
Joung, J. Keith .
NATURE, 2018, 561 (7723) :416-+
[3]   Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease [J].
Anders, Carolin ;
Niewoehner, Ole ;
Duerst, Alessia ;
Jinek, Martin .
NATURE, 2014, 513 (7519) :569-+
[4]   Incorporation of dUMP into DNA is a major source of spontaneous DNA damage, while excision of uracil is not required for cytotoxicity of fluoropyrimidines in mouse embryonic fibroblasts [J].
Andersen, S ;
Heine, T ;
Sneve, R ;
König, I ;
Krokan, HE ;
Epe, B ;
Nilsen, H .
CARCINOGENESIS, 2005, 26 (03) :547-555
[5]   Search-and-replace genome editing without double-strand breaks or donor DNA [J].
Anzalone, Andrew V. ;
Randolph, Peyton B. ;
Davis, Jessie R. ;
Sousa, Alexander A. ;
Koblan, Luke W. ;
Levy, Jonathan M. ;
Chen, Peter J. ;
Wilson, Christopher ;
Newby, Gregory A. ;
Raguram, Aditya ;
Liu, David R. .
NATURE, 2019, 576 (7785) :149-+
[6]   Novel Engineered Programmable Systems for ADAR-Mediated RNA Editing [J].
Aquino-Jarquin, Guillermo .
MOLECULAR THERAPY-NUCLEIC ACIDS, 2020, 19 :1065-1072
[7]   CRISPR/Cas9-mediated precise genome modification by a long ssDNA template in zebrafish [J].
Bai, Haipeng ;
Liu, Lijun ;
An, Ke ;
Lu, Xiaochan ;
Harrison, Michael ;
Zhao, Yanqiu ;
Yan, Ruibin ;
Lu, Zhijie ;
Li, Song ;
Lin, Shuo ;
Liang, Fang ;
Qin, Wei .
BMC GENOMICS, 2020, 21 (01)
[8]   Lessons from Enzyme Kinetics Reveal Specificity Principles for RNA-Guided Nucleases in RNA Interference and CRISPR-Based Genome Editing [J].
Bisaria, Namita ;
Jarmoskaite, Inga ;
Herschlag, Daniel .
CELL SYSTEMS, 2017, 4 (01) :21-29
[9]   Bacteriophage genes that inactivate the CRISPR/Cas bacterial immune system [J].
Bondy-Denomy, Joe ;
Pawluk, April ;
Maxwell, Karen L. ;
Davidson, Alan R. .
NATURE, 2013, 493 (7432) :429-U181
[10]   CRISPR-induced double-strand breaks trigger recombination between homologous chromosome arms [J].
Brunner, Erich ;
Yagi, Ryohei ;
Debrunner, Marc ;
Beck-Schneider, Dezirae ;
Burger, Alexa ;
Escher, Eliane ;
Mosimann, Christian ;
Hausmann, George ;
Basler, Konrad .
LIFE SCIENCE ALLIANCE, 2019, 2 (03)
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