More precise, more universal and more specific - the next generation of RNA-guided endonucleases for genome editing.

Publisher: Published by Blackwell Pub. on behalf of the Federation of European Biochemical Societies Country of Publication: England NLM ID: 101229646 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1742-4658 (Electronic) Linking ISSN: 1742464X NLM ISO Abbreviation: FEBS J Subsets: MEDLINE

Original Publication: Oxford, UK : Published by Blackwell Pub. on behalf of the Federation of European Biochemical Societies, c2005-

Gene Editing*; Clustered Regularly Interspaced Short Palindromic Repeats/*genetics ; RNA, Guide, CRISPR-Cas Systems/*genetics; Animals ; DNA/genetics ; Endonucleases/genetics ; Endonucleases/metabolism ; Humans

CRISPR is a prokaryotic defence system that was adapted as a tool for genome editing and has become one of the most important discoveries of this century. CRISPR-associated endonucleases cleave DNA at precise sites, which are marked by complementary short-guided RNA. The recently developed versions of endonucleases are compatible with a broad range of PAM motifs, have a higher specificity and enable a specific nucleotide to be replaced.
(© 2019 Federation of European Biochemical Societies.)

Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA & Charpentier EA (2012) Programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816-821.
Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI & Liu DR (2017) Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage. Nature 551, 464-471.
Komor AC, Kim YB, Packer MS, Zuris JA & Liu DR (2016) Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 533, 420-424.
Kuscu C, Parlak M, Tufan T, Yang J, Szlachta K, Wei X, Mammadov R & Adli M (2017) CRISPR-STOP: gene silencing through base-editing-induced nonsense mutations. Nat Methods 14, 710-712.
Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI & Liu DR (2017) Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage. Nature 551, 464-471.
Li C, Zong Y, Wang Y, Jin S, Zhang D, Song Q, Zhang R & Gao C (2018) Expanded base editing in rice and wheat using a Cas9-adenosine deaminase fusion. Genome Biol 19, 59.
Lu Y & Zhu JK (2017) Precise editing of a target base in the rice genome using a modified CRISPR/Cas9 system. Mol Plant 10, 523-525.
Zong Y, Wang Y, Li C, Zhang R, Chen K, Ran Y, Qiu JL, Wang D & Gao C (2017) Precise base editing in rice, wheat and maize with a Cas9-cytidine deaminase fusion. Nat Biotechnol 35, 438-440.
Qin L, Li J, Wang Q, Xu Z, Sun L, Alariqi M, Manghwar H, Wang G, Li B, Ding X et al. (2019) High-efficient and precise base editing of C•G to T•A in the allotetraploid cotton (Gossypium hirsutum) genome using a modified CRISPR/Cas9 system. Plant Biotechnol J. https://doi.org/10.1111/pbi.13168.
Mishra R, Joshi RK & Zhao K (2019) Base editing in crops: current advances, limitations and future implications. Plant Biotechnol J. https://doi.org/10.1111/pbi.13225.
Zhang Y, Malzahn AA, Sretenovic S & Qi Y (2019) The emerging and uncultivated potential of CRISPR technology in plant science. Nat Plants 5, 778-794.
Kim K, Ryu SM, Kim ST, Baek G, Kim D, Lim K, Chung E, Kim S & Kim JS (2017) Highly efficient RNA-guided base editing in mouse embryos. Nat Biotechnol 35, 435-437.
Jiang W, Feng S, Huang S, Yu W, Li G, Yang G, Liu Y, Zhang Y, Zhang L, Hou Y et al. (2018) BE-PLUS: a new base editing tool with broadened editing window and enhanced fidelity. Cell Res 28, 855-861.
Tan J, Zhang F, Karcher D & Bock R (2019) Engineering of high-precision base editors for site-specific single nucleotide replacement. Nat Commun 10, 439.
Zhang Y, Ge X, Yang F, Zhang L, Zheng J, Tan X, Jin ZB, Qu J & Gu F (2014) Comparison of non-canonical PAMs for CRISPR/Cas9-mediated DNA cleavage in human cells. Sci Rep 4, 5405.
O'Geen H, Henry IM, Bhakta MS, Meckler JF & Segal DJ (2015) A genome-wide analysis of Cas9 binding specificity using ChIP-seq and targeted sequence capture. Nucleic Acids Res 43, 3389-3404.
Hu JH, Miller SM, Geurts MH, Tang W, Chen L, Sun N, Zeina CM, Gao X, Rees HA, Lin Z et al. (2018) Evolved Cas9 variants with broad PAM compatibility and high DNA specificity. Nature 556, 57-63.
Nishimasu H, Shi X, Ishiguro S, Gao L, Hirano S, Okazaki S, Noda T, Abudayyeh OO, Gootenberg JS, Mori H et al. (2018) Engineered CRISPR-Cas9 nuclease with expanded targeting space. Science 361, 1259-1262.
Fu Y, Foden JA, Khayter C, Maeder ML, Reyon D, Joung JK & Sander JD (2013) High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol 31, 822-826.
Cho SW, Kim S, Kim Y, Kweon J, Kim HS, Bae S & Kim JS (2014) Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Genome Res 24, 132-141.
Wu X, Scott DA, Kriz AJ, Chiu AC, Hsu PD, Dadon DB, Cheng AW, Trevino AE, Konermann S, Chen S et al. (2014) Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells. Nat Biotechnol 32, 670-676.
Tsai SQ, Zheng Z, Nguyen NT, Liebers M, Topkar VV, Thapar V, Wyvekens N, Khayter C, Iafrate AJ, Le LP et al. (2015) GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol 33, 187-197.
Kim D, Bae S, Park J, Kim E, Kim S, Yu HR, Hwang J, Kim JI & Kim JS (2015) Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells. Nat Methods 12, 237-243.
Kim S, Kim D, Cho SW, Kim J & Kim JS (2014) Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res 24, 1012-1019.
Kleinstiver BP, Pattanayak V, Prew MS, Tsai SQ, Nguyen NT, Zheng Z & Joung JK (2016) High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects. Nature 529, 490-495.
Slaymaker IM, Gao L, Zetsche B, Scott DA, Yan WX & Zhang F (2016) Rationally engineered Cas9 nucleases with improved specificity. Science 351, 84-88.
Yin H, Song CQ, Suresh S, Kwan SY, Wu Q, Walsh S, Ding J, Bogorad RL, Zhu LJ, Wolfe SA et al. (2018) Anderson DG Partial DNA-guided Cas9 enables genome editing with reduced off-target activity. Nat Chem Biol 14, 311-316.
Rees HA, Wilson C, Doman JL & Liu DR (2019) Analysis and minimization of cellular RNA editing by DNA adenine base editors. Sci Adv 5, 5717.
Cox DBT, Gootenberg JS, Abudayyeh OO, Franklin B, Kellner MJ, Joung J & Zhang F (2017) RNA editing with CRISPR-Cas13. Science 358, 1019-1027.
Adli M (2018) The CRISPR tool kit for genome editing and beyond. Nat Commun 9, 1911.

FKZ 031B0547 International German Federal Ministry of Education and Research; CZ.02.1.01./0.0/0.0/16_019/0000827 International Czech Science Foundation; SPP 813103381 International Czech Science Foundation

0 (RNA, Guide, CRISPR-Cas Systems)
9007-49-2 (DNA)
EC 3.1.- (Endonucleases)

Date Created: 20191016 Date Completed: 20200615 Latest Revision: 20240104

20240104

10.1111/febs.15079

31612609