Recent application of CRISPR-Cas12 and OMEGA system for genome editing

被引：30
作者：
Badon, Isabel Wen [1 ]
Oh, Yeounsun [1 ]
Kim, Ho-Joong [2 ]
Lee, Seung Hwan [1 ]
System, Omega effector
机构：
[1] Chung Ang Univ, Dept Life Sci, Seoul 06974, South Korea
[2] Chosun Univ, Dept Chem Educ, Kwangju 61452, South Korea
来源：
MOLECULAR THERAPY | 2024年 / 32卷 / 01期
关键词：
RNA-GUIDED ENDONUCLEASE; TARGET DNA RECOGNITION; R-LOOP COMPLEX; STRUCTURAL BASIS; CRYSTAL-STRUCTURE; PAM RECOGNITION; CAS SYSTEMS; CPF1; CLEAVAGE; BASE;
D O I：
10.1016/j.ymthe.2023.11.013
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
In 2012, it was discovered that precise gene editing could be induced in target DNA using the reprogrammable characteristics of the CRISPR system. Since then, several studies have investigated the potential of the CRISPR system to edit various biological organisms. For the typical CRISPR system obtained from bacteria and archaea, many application studies have been conducted and have spread to various fields. To date, orthologs with various characteristics other than CRISPR-Cas9 have been discovered and are being intensively studied in the field of gene editing. CRISPR-Cas12 and its varied orthologs are representative examples of genome editing tools and have superior properties in terms of in vivo target gene editing compared with Cas9. Recently, TnpB and Fanzor of the OMEGA (obligate mobile element guided activity) system were identified to be the ancestor of CRISPR-Cas12 on the basis of phylogenetic analysis. Notably, the compact sizes of Cas12 and OMEGA endonucleases allow adeno-associated virus (AAV) delivery; hence, they are set to challenge Cas9 for in vivo gene therapy. This review is focused on these RNA-guided reprogrammable endonucleases: their structure, biochemistry, off-target effects, and applications in therapeutic gene editing.
引用
收藏
页码：32 / 43
页数：12
相关论文
共 114 条
[91]   The Versatile Type V CRISPR Effectors and Their Application Prospects [J].
Tong, Baisong ;
Dong, Huina ;
Cui, Yali ;
Jiang, Pingtao ;
Jin, Zhaoxia ;
Zhang, Dawei .
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY, 2021, 8
[92]   Programmable A-to-Y base editing by fusing an adenine base editor with an N-methylpurine DNA glycosylase [J].
Tong, Huawei ;
Wang, Xuchen ;
Liu, Yuanhua ;
Liu, Nana ;
Li, Yun ;
Luo, Jiamin ;
Ma, Qian ;
Wu, Danni ;
Li, Jiyong ;
Xu, Chunlong ;
Yang, Hui .
NATURE BIOTECHNOLOGY, 2023, 41 (08) :1080-+
[93]   Mb- and FnCpf1 nucleases are active in mammalian cells: activities and PAM preferences of four wild-type Cpf1 nucleases and of their altered PAM specificity variants [J].
Toth, Eszter ;
Czene, Bernadett C. ;
Kulcsar, Peter I. ;
Krausz, Sarah L. ;
Talas, Andras ;
Nyeste, Antal ;
Varga, Eva ;
Huszar, Krisztina ;
Weinhardt, Nora ;
Ligeti, Zoltan ;
Borsy, Adrienn E. ;
Fodor, Elfrieda ;
Welker, Ervin .
NUCLEIC ACIDS RESEARCH, 2018, 46 (19) :10272-10285
[94]   GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases [J].
Tsai, Shengdar Q. ;
Zheng, Zongli ;
Nguyen, Nhu T. ;
Liebers, Matthew ;
Topkar, Ved V. ;
Thapar, Vishal ;
Wyvekens, Nicolas ;
Khayter, Cyd ;
Iafrate, A. John ;
Le, Long P. ;
Aryee, Martin J. ;
Joung, J. Keith .
NATURE BIOTECHNOLOGY, 2015, 33 (02) :187-197
[95]   Highly efficient homology-directed repair using CRISPR/Cpf1-geminiviral replicon in tomato [J].
Vu, Tien Van ;
Sivankalyani, Velu ;
Kim, Eun-Jung ;
Doan, Duong Thi Hai ;
Tran, Mil Thi ;
Kim, Jihae ;
Sung, Yeon Woo ;
Park, Minwoo ;
Kang, Yang Jae ;
Kim, Jae-Yean .
PLANT BIOTECHNOLOGY JOURNAL, 2020, 18 (10) :2133-2143
[96]   Adeno-associated virus vector as a platform for gene therapy delivery [J].
Wang, Dan ;
Tai, Phillip W. L. ;
Gao, Guangping .
NATURE REVIEWS DRUG DISCOVERY, 2019, 18 (05) :358-378
[97]   A highly specific CRISPR-Cas12j nuclease enables allele- specific genome editing [J].
Wang, Yao ;
Qi, Tao ;
Liu, Jingtong ;
Yang, Yuan ;
Wang, Ziwen ;
Wang, Ying ;
Wang, Tianyi ;
Li, Miaomiao ;
Li, Mingqing ;
Lu, Daru ;
Chang, Alex Chia Yu ;
Yang, Li ;
Gao, Song ;
Wang, Yongming ;
Lan, Feng .
SCIENCE ADVANCES, 2023, 9 (06)
[98]   Cas12c-DETECTOR: A specific and sensitive Cas12c-based DNA detection platform [J].
Wang, Zupeng ;
Zhong, Caihong .
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES, 2021, 193 :441-449
[99]   An engineered hypercompact CRISPR-Cas12f system with boosted gene-editing activity [J].
Wu, Tong ;
Liu, Chang ;
Zou, Siyuan ;
Lyu, Ruitu ;
Yang, Bowei ;
Yan, Hao ;
Zhao, Minglei ;
Tang, Weixin .
NATURE CHEMICAL BIOLOGY, 2023, 19 (11) :1384-+
[100]   The miniature CRISPR-Cas12m effector binds DNA to block transcription [J].
Wu, Wen Y. ;
Mohanraju, Prarthana ;
Liao, Chunyu ;
Adiego-Perez, Belen ;
Creutzburg, Sjoerd C. A. ;
Makarova, Kira S. ;
Keessen, Karlijn ;
Lindeboom, Timon A. ;
Khan, Tahseen S. ;
Prinsen, Stijn ;
Joosten, Rob ;
Yan, Winston X. ;
Migur, Anzhela ;
Laffeber, Charlie ;
Scott, David A. ;
Lebbink, Joyce H. G. ;
Koonin, Eugene, V ;
Beisel, Chase L. ;
van der Oost, John .
MOLECULAR CELL, 2022, 82 (23) :4487-+
3456789101112