Past, present, and future of CRISPR genome editing technologies

被引：189

作者：

Pacesa, Martin ^{[1
,2
]}

Pelea, Oana ^{[3
]}

Jinek, Martin ^{[3
]}

机构：

[1] Ecole Polytech Fed Lausanne, Lab Prot Design & Immunoengn, Stn 19, CH-1015 Lausanne, Switzerland

[2] Swiss Inst Bioinformat, Stn 19, CH-1015 Lausanne, Switzerland

[3] Univ Zurich, Dept Biochem, Winterthurerstr 190, CH-8057 Zurich, Switzerland

来源：

CELL | 2024年 / 187卷 / 05期

基金：

欧洲研究理事会; 瑞士国家科学基金会;

关键词：

RNA-GUIDED ENDONUCLEASE; ALTERED PAM SPECIFICITIES; HOMOLOGY-DIRECTED REPAIR; NUCLEIC-ACID DETECTION; OFF-TARGET ACTIVITY; STRUCTURAL BASIS; DNA CLEAVAGE; WIDE ANALYSIS; CAS SYSTEMS; HUMAN-CELLS;

D O I：

10.1016/j.cell.2024.01.042

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Genome editing has been a transformative force in the life sciences and human medicine, offering unprecedented opportunities to dissect complex biological processes and treat the underlying causes of many genetic diseases. CRISPR-based technologies, with their remarkable efficiency and easy programmability, stand at the forefront of this revolution. In this Review, we discuss the current state of CRISPR gene editing technologies in both research and therapy, highlighting limitations that constrain them and the technological innovations that have been developed in recent years to address them. Additionally, we examine and summarize the current landscape of gene editing applications in the context of human health and therapeutics. Finally, we outline potential future developments that could shape gene editing technologies and their applications in the coming years.

引用

页码：1076 / 1100

页数：25

共 344 条

[1] A cytosine deaminase for programmable single-base RNA editing [J].

Abudayyeh, Omar O. ;

Gootenberg, Jonathan S. ;

Franklin, Brian ;

Koob, Jeremy ;

Kellner, Max J. ;

Ladha, Alim ;

Joung, Julia ;

Kirchgatterer, Paul ;

Cox, David B. T. ;

Zhang, Feng .

SCIENCE, 2019, 365 (6451) :382-+

[2] C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector [J].

Abudayyeh, Omar O. ;

Gootenberg, Jonathan S. ;

Konermann, Silvana ;

Joung, Julia ;

Slaymaker, Ian M. ;

Cox, David B. T. ;

Shmakov, Sergey ;

Makarova, Kira S. ;

Semenova, Ekaterina ;

Minakhin, Leonid ;

Severinov, Konstantin ;

Regev, Aviv ;

Lander, Eric S. ;

Koonin, Eugene V. ;

Zhang, Feng .

SCIENCE, 2016, 353 (6299)

[3] Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors [J].

Al-Shayeb, Basem ;

Skopintsev, Petr ;

Soczek, Katarzyna M. ;

Stahl, Elizabeth C. ;

Li, Zheng ;

Groover, Evan ;

Smock, Dylan ;

Eggers, Amy R. ;

Pausch, Patrick ;

Cress, Brady F. ;

Huang, Carolyn J. ;

Staskawicz, Brian ;

Savage, David F. ;

Jacobsen, Steven E. ;

Banfield, Jillian F. ;

Doudna, Jennifer A. .

CELL, 2022, 185 (24) :4574-+

[4] Whole-organism clone tracing using single-cell sequencing [J].

Alemany, Anna ;

Florescu, Maria ;

Baron, Chloe S. ;

Peterson-Maduro, Josi ;

van Oudenaarden, Alexander .

NATURE, 2018, 556 (7699) :108-+

[5] Electroporation and genetic supply of Cas9 increase the generation efficiency of CRISPR/Cas9 knock-in alleles in C57BL/6J mouse zygotes [J].

Alghadban, Samy ;

Bouchareb, Amine ;

Hinch, Robert ;

Hernandez-Pliego, Polinka ;

Biggs, Daniel ;

Preece, Chris ;

Davies, Benjamin .

SCIENTIFIC REPORTS, 2020, 10 (01)

[6] Uncovering the functional diversity of rare CRISPR-Cas systems with deep terascale clustering [J].

Altae-Tran, Han ;

Kannan, Soumya ;

Suberski, Anthony J. ;

Mears, Kepler S. ;

Demircioglu, F. Esra ;

Moeller, Lukas ;

Kocalar, Selin ;

Oshiro, Rachel ;

Makarova, Kira S. ;

Macrae, Rhiannon K. ;

Koonin, Eugene V. ;

Zhang, Feng .

SCIENCE, 2023, 382 (6673)

[7] The widespread IS200/IS605 transposon family encodes diverse programmable RNA-guided endonucleases [J].

Altae-Tran, Han ;

Kannan, Soumya ;

Demircioglu, F. Esra ;

Oshiro, Rachel ;

Nety, Suchita P. ;

McKay, Luke J. ;

Dlakic, Mensur ;

Inskeep, William P. ;

Makarova, Kira S. ;

Macrae, Rhiannon K. ;

Koonin, Eugene, V ;

Zhang, Feng .

SCIENCE, 2021, 374 (6563) :57-+

[8] Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy [J].

Amoasii, Leonela ;

Hildyard, John C. W. ;

Li, Hui ;

Sanchez-Ortiz, Efrain ;

Mireault, Alex ;

Caballero, Daniel ;

Harron, Rachel ;

Stathopoulou, Thaleia-Rengina ;

Massey, Claire ;

Shelton, John M. ;

Bassel-Duby, Rhonda ;

Piercy, Richard J. ;

Olson, Eric N. .

SCIENCE, 2018, 362 (6410) :86-90

[9] Structural Plasticity of PAM Recognition by Engineered Variants of the RNA-Guided Endonuclease Cas9 [J].

Anders, Carolin ;

Bargsten, Katja ;

Jinek, Martin .

MOLECULAR CELL, 2016, 61 (06) :895-902

[10] Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing [J].

Anzalone, Andrew, V ;

Gao, Xin D. ;

Podracky, Christopher J. ;

Nelson, Andrew T. ;

Koblan, Luke W. ;

Raguram, Aditya ;

Levy, Jonathan M. ;

Mercer, Jaron A. M. ;

Liu, David R. .

NATURE BIOTECHNOLOGY, 2022, 40 (05) :731-+

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