A compact, high-accuracy Cas9 with a dinucleotide PAM for in vivo genome editing
Open accessAbstract
CRISPR-Cas9 genome editing has transformed biotechnology and therapeutics. However, in vivo applications of some Cas9s are hindered by large size (limiting delivery by adeno-associated virus [AAV] vectors), off-target editing, or complex protospacer-adjacent motifs (PAMs) that restrict the density of recognition sequences in target DNA. Here, we exploited natural variation in the PAM-interacting domains (PIDs) of closely related Cas9s to identify a compact ortholog from Neisseria meningitidis-Nme2Cas9-that recognizes a simple dinucleotide PAM (N 4 CC) that provides for high target site density. All-in-one AAV delivery of Nme2Cas9 with a guide RNA targeting Pcsk9 in adult mouse liver produces efficient genome editing and reduced serum cholesterol with exceptionally high specificity. We further expand our single-AAV platform to pre-implanted zygotes for streamlined generation of genome-edited mice. Nme2Cas9 combines all-in-one AAV compatibility, exceptional editing accuracy within cells, and high target site density for in vivo genome editing applications.
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The requirement of a specific PAM sequence for targeted genome editing is crucial, and even though it reduces off-target activity, it often results in suboptimal DNA cleavage and editing efficiencies and restricts the scope of targeted editing.
“Also, the requirement of a specific PAM Sequence for targeted genome editing is crucial, and even though it reduces off-target activity, it often results in suboptimal DNA cleavage, editing efficiencies (Ran et al., 2015; Edraki et al., 2019) and restricting the scope of targeted edition.”
From Emerging applications of gene editing technologies for the development of climate-resilient crops (2025) · cites this paper as Edraki et al., 2019
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Cites
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116 · see citing works
Multiplex Genome Engineering Using CRISPR/Cas Systems
Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P. D., Wu, X., Jiang, W., Marraffini, L. A., et al. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339 · see citing works
RNA-Guided Human Genome Engineering via Cas9
Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M. (2013) · Science · doi:10.1126/science.1232033 · Cited by 9,210 · see citing works
CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes
Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., Romero, D. A., & Horvath, P. (2007) · Science · doi:10.1126/science.1138140 · Cited by 6,304 · see citing works
Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299 · see citing works
Efficient genome editing in zebrafish using a CRISPR-Cas system
Hwang, W. Y., Fu, Y., Reyon, D., Maeder, M. L., Tsai, S. Q., Sander, J. D., Peterson, R. T., Yeh, J.-R. J., & Joung, J. K. (2013) · Nature Biotechnology · doi:10.1038/nbt.2501 · Cited by 2,937 · see citing works
Gasiunas, G., Barrangou, R., Horvath, P., & Siksnys, V. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1208507109 · Cited by 2,769 · see citing works
In vivo genome editing using Staphylococcus aureus Cas9
Ran, F. A., Cong, L., Yan, W. X., Scott, D. A., Gootenberg, J. S., Kriz, A. J., Zetsche, B., Shalem, O., Wu, X., Makarova, K. S., et al. (2015) · Nature · doi:10.1038/nature14299 · Cited by 2,687 · see citing works
Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes
Brouns, S. J. J., Jore, M. M., Lundgren, M., Westra, E. R., Slijkhuis, R. J. H., Snijders, A. P., Dickman, M. J., Makarova, K. S., Koonin, E. V., & John van der, O. O. S. T. (2008) · Science · doi:10.1126/science.1159689 · Cited by 2,593 · see citing works
CRISPR RNA Maturation by Trans-Encoded Small RNA and Host Factor RNase III
Deltcheva, E., Chylinski, K., Sharma, C. M., Gonzales, K., Chao, Y., Pirzada, Z. A., Eckert, M. R., Vogel, J., & Charpentier, E. (2011) · Nature · doi:10.1038/nature09886 · Cited by 2,563 · see citing works
RNA-guided editing of bacterial genomes using CRISPR-Cas systems
Jiang, W., Bikard, D., Cox, D., Zhang, F., & Marraffini, L. A. (2013) · Nature Biotechnology · doi:10.1038/nbt.2508 · Cited by 2,429 · see citing works
Easy quantitative assessment of genome editing by sequence trace decomposition
Brinkman, E. K., Chen, T., Amendola, M., & van Steensel, B. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku936 · Cited by 2,390 · see citing works
The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA
Garneau, J. E., Dupuis, M.-È., Villion, M., Romero, D. A., Barrangou, R., Boyaval, P., Fremaux, C., Horvath, P., Magadán, A. H., & Moineau, S. (2010) · Nature · doi:10.1038/nature09523 · Cited by 2,336 · see citing works
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
Tsai, S. Q., Zheng, Z., Nguyen, N. T., Liebers, M., Topkar, V. V., Thapar, V., Wyvekens, N., Khayter, C., Iafrate, A. J., Le, L. P., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3117 · Cited by 2,268 · see citing works
RNA-programmed genome editing in human cells
Jinek, M., East, A., Cheng, A., Lin, S., Ma, E., & Doudna, J. (2013) · eLife · doi:10.7554/elife.00471 · Cited by 2,098 · see citing works
Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
Fu, Y., Sander, J. D., Reyon, D., Cascio, V. M., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2808 · Cited by 1,993 · cited 1× · see citing works
“For off-target identification, the analysis revealed that the DS2, DS4, and DS6 SpyCas9 sgRNAs appeared to direct editing at 93, 10, and 118 candidate off-target sites, respectively, within the normal range of GUIDE-seq off-targets with plasmid-based SpyCas9 editing (Fu et al., 2014; Tsai et al., 2014).”
BackgroundTargeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
Cho, S. W., Kim, S., Kim, J. M., & Kim, J.-S. (2013) · Nature Biotechnology · doi:10.1038/nbt.2507 · Cited by 1,863 · see citing works
Kim, S., Kim, D., Cho, S. W., Kim, J., & Kim, J.-S. (2014) · Genome Research · doi:10.1101/gr.171322.113 · Cited by 1,856 · see citing works
Mali, P., Aach, J., Stranges, P. B., Esvelt, K. M., Moosburner, M., Kosuri, S., Yang, L., & Church, G. M. (2013) · Nature Biotechnology · doi:10.1038/nbt.2675 · Cited by 1,752 · cited 1× · see citing works
“SpyCas9 nickases (nCas9s) have been used to induce homology-directed repair (HDR) and to improve genome editing specificity via DSB induction by dual nickases (Mali et al., 2013a; Ran et al., 2013).”
BackgroundCRISPR Interference Limits Horizontal Gene Transfer in Staphylococci by Targeting DNA
Marraffini, L. A., & Sontheimer, E. J. (2008) · Science · doi:10.1126/science.1165771 · Cited by 1,752 · see citing works
Base editing: precision chemistry on the genome and transcriptome of living cells
Rees, H. A., & Liu, D. R. (2018) · Nature Reviews Genetics · doi:10.1038/s41576-018-0059-1 · Cited by 1,675 · see citing works
Evolved Cas9 variants with broad PAM compatibility and high DNA specificity
Hu, J. H., Miller, S. M., Geurts, M. H., Tang, W., Chen, L., Sun, N., Zeina, C. M., Gao, X., Rees, H. A., Lin, Z., et al. (2018) · Nature · doi:10.1038/nature26155 · Cited by 1,552 · see citing works
Short motif sequences determine the targets of the prokaryotic CRISPR defence system
Mojica, F. J. M., Díez‐Villaseñor, C., García‐Martínez, J., & Almendros, C. (2009) · Microbiology · doi:10.1099/mic.0.023960-0 · Cited by 1,500 · see citing works
Zuris, J. A., Thompson, D. B., Shu, Y., Guilinger, J. P., Bessen, J. L., Hu, J. H., Maeder, M. L., Joung, J. K., Chen, Z.-Y., & Liu, D. R. (2015) · Nature Biotechnology · doi:10.1038/nbt.3081 · Cited by 1,460 · see citing works
Friedrich, G., & Soriano, P. M. (1991) · Genes & Development · doi:10.1101/gad.5.9.1513 · Cited by 1,403 · see citing works
The Liver as an Immunological Organ
Racanelli, V., & Rehermann, B. (2006) · Hepatology · doi:10.1002/hep.21060 · Cited by 1,377 · see citing works
Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
Cho, S. W., Kim, S., Kim, Y., Kweon, J., Kim, H. S., Bae, S., & Kim, J.-S. (2014) · Genome Research · doi:10.1101/gr.162339.113 · Cited by 1,356 · see citing works
Phage Response to CRISPR-Encoded Resistance in Streptococcus thermophilus
Deveau, H., Barrangou, R., Garneau, J. E., Labonté, J., Fremaux, C., Boyaval, P., Romero, D. A., Horvath, P., & Moineau, S. (2007) · Journal of Bacteriology · doi:10.1128/jb.01412-07 · Cited by 1,196 · see citing works
Enhanced proofreading governs CRISPR-Cas9 targeting accuracy
Chen, J. S., Dagdas, Y. S., Kleinstiver, B. P., Welch, M. M., Sousa, A. A., Harrington, L. B., Sternberg, S. H., Joung, J. K., Yildiz, A., & Doudna, J. A. (2017) · Nature · doi:10.1038/nature24268 · Cited by 1,168 · see citing works
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
Komor, A. C., Badran, A. H., & Liu, D. R. (2016) · Cell · doi:10.1016/j.cell.2016.10.044 · Cited by 1,011 · see citing works
The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli
Sapranauskas, R., Gasiūnas, G., Fremaux, C., Barrangou, R., Horvath, P., & Šikšnys, V. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr606 · Cited by 835 · see citing works
Beyond editing: repurposing CRISPR-Cas9 for precision genome regulation and interrogation
Dominguez, A. A., Lim, W. A., & Qi, L. S. (2016) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2015.2 · Cited by 832 · cited 1× · see citing works
“The availability of a catalytically dead version of Nme2Cas9 (dNme2Cas9) also promises to expand the scope of applications such as CRISPRi, CRISPRa, base editing, and related approaches (Dominguez et al., 2016; Komor et al., 2017).”
BackgroundOrthogonal Cas9 proteins for RNA-guided gene regulation and editing
Esvelt, K. M., Mali, P., Braff, J. L., Moosburner, M., Yaung, S. J., & Church, G. M. (2013) · Nature Methods · doi:10.1038/nmeth.2681 · Cited by 786 · see citing works
In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni
Kim, E., Koo, T., Park, S. W., Kim, D., Kim, K., Cho, H.-Y., Song, D. W., Lee, K. J., Jung, M. H., Kim, S., et al. (2017) · Nature Communications · doi:10.1038/ncomms14500 · Cited by 715 · see citing works
Generation of knock-in primary human T cells using Cas9 ribonucleoproteins
Schumann, K., Lin, S., Boyer, E., Simeonov, D. R., Subramaniam, M., Gate, R. E., Haliburton, G. E., Ye, C. J., Bluestone, J. A., Doudna, J. A., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1512503112 · Cited by 702 · see citing works
Hou, Z., Zhang, Y., Propson, N. E., Howden, S. E., Chu, L.-F., Sontheimer, E. J., & Thomson, J. A. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1313587110 · Cited by 681 · see citing works
Decreased plasma cholesterol and hypersensitivity to statins in mice lacking <i>Pcsk9</i>
Rashid, S., Curtis, D. E., Garuti, R., Anderson, N. N., Bashmakov, Y., Ho, Y. K., Hammer, R. E., Moon, Y.-A., & Horton, J. D. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0501652102 · Cited by 679 · see citing works
Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition
Kleinstiver, B. P., Prew, M. S., Tsai, S. Q., Nguyen, N. T., Topkar, V. V., Zheng, Z., & Joung, J. K. (2015) · Nature Biotechnology · doi:10.1038/nbt.3404 · Cited by 611 · see citing works
Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases
Tsai, S. Q., & Joung, J. K. (2016) · Nature Reviews Genetics · doi:10.1038/nrg.2016.28 · Cited by 508 · see citing works
A highly specific SpCas9 variant is identified by in vivo screening in yeast
Casini, A., Olivieri, M., Petris, G., Montagna, C., Reginato, G., Maule, G., Lorenzin, F., Prandi, D., Romanel, A., Demichelis, F., et al. (2018) · Nature Biotechnology · doi:10.1038/nbt.4066 · Cited by 493 · see citing works
Inhibition of CRISPR-Cas9 with Bacteriophage Proteins
Rauch, B. J., Silvis, M. R., Hultquist, J. F., Waters, C. S., McGregor, M., Krogan, N. J., & Bondy‐Denomy, J. (2016) · Cell · doi:10.1016/j.cell.2016.12.009 · Cited by 454 · see citing works
Analyzing CRISPR genome-editing experiments with CRISPResso
Pinello, L., Canver, M. C., Hoban, M. D., Orkin, S. H., Kohn, D. B., Bauer, D. E., & Yuan, G. (2016) · Nature Biotechnology · doi:10.1038/nbt.3583 · Cited by 450 · see citing works
Naturally Occurring Off-Switches for CRISPR-Cas9
Pawluk, A., Amrani, N., Zhang, Y., Garcı́a, B., Hidalgo-Reyes, Y., Lee, J., Edraki, A., Shah, M. K., Sontheimer, E. J., Maxwell, K. L., et al. (2016) · Cell · doi:10.1016/j.cell.2016.11.017 · Cited by 384 · see citing works
Disabling Cas9 by an anti-CRISPR DNA mimic
Shin, J. J., Jiang, F., Liu, J., Bray, N., Rauch, B. J., Baik, R., Nogales, E., Bondy‐Denomy, J., Corn, J. E., & Doudna, J. A. (2017) · Science Advances · doi:10.1126/sciadv.1701620 · Cited by 351 · see citing works
Fonfara, I., Rhun, A. L., Chylinski, K., Makarova, K. S., Lécrivain, A.-L., Bzdrenga, J., Koonin, E. V., & Charpentier, E. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt1074 · Cited by 329 · see citing works
Tracking genome engineering outcome at individual DNA breakpoints
Certo, M. T., Ryu, B. Y., Annis, J. E., Garibov, M., Jarjour, J., Rawlings, D. J., & Scharenberg, A. M. (2011) · Nature Methods · doi:10.1038/nmeth.1648 · Cited by 309 · see citing works
Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity
Tycko, J., Myer, V. E., & Hsu, P. D. (2016) · Molecular Cell · doi:10.1016/j.molcel.2016.07.004 · Cited by 304 · see citing works
Processing-Independent CRISPR RNAs Limit Natural Transformation in Neisseria meningitidis
Zhang, Y., Heidrich, N., Ampattu, B. J., Gunderson, C. W., Seifert, H. S., Schoen, C. U., Vogel, J., & Sontheimer, E. J. (2013) · Molecular Cell · doi:10.1016/j.molcel.2013.05.001 · Cited by 283 · see citing works
Friedland, A. E., Baral, R., Singhal, P., Loveluck, K., Shen, S., Sanchez, M. E., Marco, E., Gotta, G. M., Maeder, M. L., Kennedy, E. M., et al. (2015) · Genome biology · doi:10.1186/s13059-015-0817-8 · Cited by 280 · see citing works
Pawluk, A., Bondy‐Denomy, J., Cheung, V. H. W., Maxwell, K. L., & Davidson, A. R. (2014) · mBio · doi:10.1128/mbio.00896-14 · Cited by 263 · see citing works
Systematic gene tagging using CRISPR/Cas9 in human stem cells to illuminate cell organization
Roberts, B., Haupt, A., Tucker, A., Grancharova, T., Arakaki, J., Fuqua, M. A., Nelson, A., Hookway, C., Ludmann, S. A., Mueller, I. A., et al. (2017) · Molecular Biology of the Cell · doi:10.1091/mbc.e17-03-0209 · Cited by 262 · see citing works
Structural basis of CRISPR–SpyCas9 inhibition by an anti-CRISPR protein
Dong, D., Guo, M., Wang, S., Zhu, Y., Wang, S., Xiong, Z., Yang, J., Xu, Z., & Huang, Z. (2017) · Nature · doi:10.1038/nature22377 · Cited by 255 · see citing works
A Broad-Spectrum Inhibitor of CRISPR-Cas9
Harrington, L. B., Doxzen, K. W., Ma, E., Liu, J., Knott, G. J., Edraki, A., Garcı́a, B., Amrani, N., Chen, J. S., Cofsky, J. C., et al. (2017) · Cell · doi:10.1016/j.cell.2017.07.037 · Cited by 254 · see citing works
The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells
Lee, C. M., Cradick, T. J., & Bao, G. (2016) · Molecular Therapy · doi:10.1038/mt.2016.8 · Cited by 244 · see citing works
Partial DNA-guided Cas9 enables genome editing with reduced off-target activity
Yin, H., Song, C.-Q., Suresh, S., Kwan, S.-Y., Wu, Q., Walsh, S., Ding, J., Bogorad, R. L., Zhu, L. J., Wolfe, S. A., et al. (2018) · Nature Chemical Biology · doi:10.1038/nchembio.2559 · Cited by 240 · see citing works
Rapid characterization of CRISPR-Cas9 protospacer adjacent motif sequence elements
Karvelis, T., Gasiūnas, G., Young, J. K., Bigelyte, G., Šilanskas, A., Cigan, M., & Šikšnys, V. (2015) · Genome biology · doi:10.1186/s13059-015-0818-7 · Cited by 222 · see citing works
Yokoyama, T., Silversides, D. W., Waymire, K. G., Kwon, B. S., Takeuchi, T., & Overbeek, P. A. (1990) · Nucleic Acids Research · doi:10.1093/nar/18.24.7293 · Cited by 207 · see citing works
A Thermostable Cas9 with Increased Lifetime in Human Plasma
Harrington, L. B., Paez-Espino, D., Staahl, B. T., Chen, J. S., Ma, E., Kyrpides, N. C., & Doudna, J. A. (2017) · Nature Communications · doi:10.1038/s41467-017-01408-4 · Cited by 205 · see citing works
CRISPR Immunity Drives Rapid Phage Genome Evolution in Streptococcus thermophilus
Páez-Espino, D., Sharon, I., Morovic, W., Stahl, B., Thomas, B. C., Barrangou, R., & Banfield, J. F. (2015) · mBio · doi:10.1128/mbio.00262-15 · Cited by 202 · see citing works
RNA-based recognition and targeting: sowing the seeds of specificity
Gorski, S. A., Vogel, J., & Doudna, J. A. (2017) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2016.174 · Cited by 191 · see citing works
Zhu, L. J., Holmes, B. R., D, N. A. M., & Brodsky, M. H. (2014) · PLoS ONE · doi:10.1371/journal.pone.0108424 · Cited by 190 · see citing works
An anti-CRISPR from a virulent streptococcal phage inhibits Streptococcus pyogenes Cas9
Hynes, A. P., Rousseau, G. M., Lemay, M.-L., Horvath, P., Romero, D. A., Fremaux, C., & Moineau, S. (2017) · Nature Microbiology · doi:10.1038/s41564-017-0004-7 · Cited by 185 · see citing works
Inhibition Mechanism of an Anti-CRISPR Suppressor AcrIIA4 Targeting SpyCas9
Yang, H., & Patel, D. J. (2017) · Molecular Cell · doi:10.1016/j.molcel.2017.05.024 · Cited by 178 · see citing works
Type II-C CRISPR-Cas9 Biology, Mechanism, and Application
Mir, A., Edraki, A., Lee, J., & Sontheimer, E. J. (2017) · ACS Chemical Biology · doi:10.1021/acschembio.7b00855 · Cited by 148 · see citing works
Gene Therapy 2017: Progress and Future Directions
Keeler, A. M., ElMallah, M. K., & Flotte, T. R. (2017) · Clinical and Translational Science · doi:10.1111/cts.12466 · Cited by 135 · see citing works
Single-Stranded DNA Cleavage by Divergent CRISPR-Cas9 Enzymes
Ma, E., Harrington, L. B., O’Connell, M. R., Zhou, K., & Doudna, J. A. (2015) · Molecular Cell · doi:10.1016/j.molcel.2015.10.030 · Cited by 125 · see citing works
NmeCas9 is an intrinsically high-fidelity genome-editing platform
Amrani, N., Gao, X. D., Liu, P., Edraki, A., Mir, A., Ibraheim, R., Gupta, A., Sasaki, K. E., Wu, T., Donohoue, P. D., et al. (2018) · Genome biology · doi:10.1186/s13059-018-1591-1 · Cited by 124 · see citing works
Zhang, Z., Theurkauf, W. E., Weng, Z., & Zamore, P. D. (2012) · Silence · doi:10.1186/1758-907x-3-9 · Cited by 122 · see citing works
DNA-binding-domain fusions enhance the targeting range and precision of Cas9
Bolukbasi, M. F., Gupta, A., Oikemus, S., Derr, A. G., Garber, M. G., Brodsky, M. H., Zhu, L. J., & Wolfe, S. A. (2015) · Nature Methods · doi:10.1038/nmeth.3624 · Cited by 114 · see citing works
Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery
Bolukbasi, M. F., Gupta, A., & Wolfe, S. A. (2015) · Nature Methods · doi:10.1038/nmeth.3684 · Cited by 111 · see citing works
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo
Ibraheim, R., Song, C., Mir, A., Amrani, N., Xue, W., & Sontheimer, E. J. (2018) · Genome biology · doi:10.1186/s13059-018-1515-0 · Cited by 110 · see citing works
Potent Cas9 Inhibition in Bacterial and Human Cells by AcrIIC4 and AcrIIC5 Anti-CRISPR Proteins
Lee, J., Mir, A., Edraki, A., Garcı́a, B., Amrani, N., Lou, H. E., Gainetdinov, I. V., Pawluk, A., Ibraheim, R., Gao, X. D., et al. (2018) · mBio · doi:10.1128/mbio.02321-18 · Cited by 99 · see citing works
Yoon, Y., Wang, D., Tai, P. W. L., Riley, J., Gao, G., & Rivera‐Pérez, J. A. (2018) · Nature Communications · doi:10.1038/s41467-017-02706-7 · Cited by 92 · see citing works
Repair of a Site-Specific DNA Cleavage: Old-School Lessons for Cas9-Mediated Gene Editing
Gallagher, D. N., & Haber, J. E. (2017) · ACS Chemical Biology · doi:10.1021/acschembio.7b00760 · Cited by 88 · see citing works
Bisaria, N., Jarmoskaite, I., & Herschlag, D. (2017) · Cell Systems · doi:10.1016/j.cels.2016.12.010 · Cited by 72 · see citing works
DNase H Activity of Neisseria meningitidis Cas9
Zhang, Y., Rajan, R., Seifert, H. S., Mondragón, A., & Sontheimer, E. J. (2015) · Molecular Cell · doi:10.1016/j.molcel.2015.09.020 · Cited by 67 · see citing works
GUIDEseq: a bioconductor package to analyze GUIDE-Seq datasets for CRISPR-Cas nucleases
Zhu, L. J., Lawrence, M., Gupta, A., Pagès, H., Küçükural, A., Garber, M. G., & Wolfe, S. A. (2017) · BMC Genomics · doi:10.1186/s12864-017-3746-y · Cited by 41 · see citing works
Potent Cas9 inhibition in bacterial and human cells by new anti-CRISPR protein familiesPreprint
Lee, J., Mir, A., Edraki, A., Garcı́a, B., Amrani, N., Lou, H. E., Gainetdinov, I. V., Pawluk, A., Ibraheim, R., Gao, X. D., et al. (2018) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/350504 · Cited by 3 · see citing works
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Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
Anzalone, A. V., Koblan, L. W., & Liu, D. R. (2020) · Nature Biotechnology · doi:10.1038/s41587-020-0561-9 · Cited by 2,237 · see citing works
CRISPR/Cas9 therapeutics: progress and prospects
Li, T., Yang, Y., Qi, H., Cui, W., Zhang, L., Fu, X., He, X., Liu, M., Li, P., & Yu, T. (2023) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-023-01309-7 · Cited by 770 · see citing works
Past, present, and future of CRISPR genome editing technologies
Pacesa, M., Pelea, O., & Jinek, M. (2024) · Cell · doi:10.1016/j.cell.2024.01.042 · Cited by 609 · see citing works
Therapeutic in vivo delivery of gene editing agents
Raguram, A., Banskota, S., & Liu, D. R. (2022) · Cell · doi:10.1016/j.cell.2022.03.045 · Cited by 532 · see citing works
Inhibition of histone deacetylase 1 (HDAC1) and HDAC2 enhances CRISPR/Cas9 genome editing
Liu, B., Chen, S., Rose, A. M. L., Chen, D., Cao, F., Zwinderman, M. R. H., Kiemel, D., Aïssi, M., Dekker, F. J., & Haisma, H. J. (2019) · Nucleic Acids Research · doi:10.1093/nar/gkz1136 · Cited by 370 · see citing works
Continuous evolution of SpCas9 variants compatible with non-G PAMs
Miller, S. M., Wang, T., Randolph, P. B., Arbab, M., Shen, M. W., Huang, T. P., Matuszek, Ż., Newby, G. A., Rees, H. A., & Liu, D. R. (2020) · Nature Biotechnology · doi:10.1038/s41587-020-0412-8 · Cited by 347 · see citing works
Keeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015530 · Cited by 322 · see citing works
CRISPR technologies and the search for the PAM-free nuclease
Collias, D., & Beisel, C. L. (2021) · Nature Communications · doi:10.1038/s41467-020-20633-y · Cited by 287 · see citing works
Neugebauer, M. E., Hsu, A., Arbab, M., Krasnow, N. A., McElroy, A. N., Pandey, S., Doman, J. L., Huang, T. P., Raguram, A., Banskota, S., et al. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01533-6 · Cited by 273 · see citing works
CRISPR technologies for genome, epigenome and transcriptome editing
Villiger, L., Joung, J., Koblan, L. W., Weissman, J. S., Abudayyeh, O. O., & Gootenberg, J. S. (2024) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-023-00697-6 · Cited by 264 · see citing works
Kumar, R., Das, S. P., Choudhury, B. U., Kumar, A., Prakash, N. R., Verma, R., Chakraborti, M., Devi, A. G., Bhattacharjee, B., Das, R., et al. (2024) · Biological Research · doi:10.1186/s40659-024-00562-6 · Cited by 271 · see citing works
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
Li, A., Tanner, M. R., Lee, C. M., Hurley, A. E., De Giorgi, M., Jarrett, K. E., Davis, T. H., Doerfler, A. M., Bao, G., Beeton, C., et al. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.04.017 · Cited by 230 · see citing works
Davis, J. R., Wang, X., Witte, I. P., Huang, T. P., Levy, J. M., Raguram, A., Banskota, S., Seidah, N. G., Musunuru, K., & Liu, D. R. (2022) · Nature Biomedical Engineering · doi:10.1038/s41551-022-00911-4 · Cited by 195 · see citing works
A catalogue of biochemically diverse CRISPR-Cas9 orthologs
Gasiūnas, G., Young, J. K., Karvelis, T., Kazlauskas, D., Urbaitis, T., Jasnauskaitė, M., Grusyte, M. M., Paulraj, S., Wang, P.-H., Hou, Z., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-19344-1 · Cited by 175 · see citing works
Drug delivery systems for CRISPR-based genome editors
Madigan, V. J., Zhang, F., & Dahlman, J. E. (2023) · Nature Reviews Drug Discovery · doi:10.1038/s41573-023-00762-x · Cited by 180 · see citing works
Anti-CRISPRs: Protein Inhibitors of CRISPR-Cas Systems
Davidson, A. R., Lu, W., Stanley, S. Y., Wang, J., Mejdani, M., Trost, C. N., Hicks, B. T., Lee, J., & Sontheimer, E. J. (2020) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-011420-111224 · Cited by 167 · see citing works
A compact Cas9 ortholog from Staphylococcus Auricularis (SauriCas9) expands the DNA targeting scope
Hu, Z., Wang, S., Zhang, C., Gao, N., Li, M., Wang, D., Wang, D., Liu, D., Liu, H., Ong, S., et al. (2020) · PLoS Biology · doi:10.1371/journal.pbio.3000686 · Cited by 142 · see citing works
Genome Engineering in Rice Using Cas9 Variants that Recognize NG PAM Sequences
Hua, K., Tao, X., Han, P., Wang, R., & Zhu, J. (2019) · Molecular Plant · doi:10.1016/j.molp.2019.03.009 · Cited by 140 · see citing works
Yamagata, T., Raveau, M., Kobayashi, K., Miyamoto, H., Tatsukawa, T., Ogiwara, I., Itohara, S., Hensch, T. K., & Yamakawa, K. (2020) · Neurobiology of Disease · doi:10.1016/j.nbd.2020.104954 · Cited by 135 · see citing works
Massively parallel kinetic profiling of natural and engineered CRISPR nucleases
Jones, S. K., Hawkins, J. A., Johnson, N. V., Jung, C., Hu, K., Rybarski, J. R., Chen, J. S., Doudna, J. A., Press, W. H., & Finkelstein, I. J. (2020) · Nature Biotechnology · doi:10.1038/s41587-020-0646-5 · Cited by 126 · see citing works
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
Wu, S., Li, Q.-C., Yin, C., Xue, W., & Song, C. (2020) · Theranostics · doi:10.7150/thno.43360 · Cited by 137 · see citing works
CRISPR–Cas applications in agriculture and plant research
Tuncel, A., Pan, C., Clem, J. S., Liu, D., & Qi, Y. (2025) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-025-00834-3 · Cited by 139 · see citing works
An engineered ScCas9 with broad PAM range and high specificity and activity
Chatterjee, P., Jakimo, N., Lee, J., Amrani, N., Rodríguez, T., Koseki, S., Tysinger, E., Qing, R., Hao, S., Sontheimer, E. J., et al. (2020) · Nature Biotechnology · doi:10.1038/s41587-020-0517-0 · Cited by 128 · see citing works
Sun, W. D., Yang, J., Cheng, Z., Amrani, N., Liu, C., Wang, K., Ibraheim, R., Edraki, A., Huang, X., Wang, M., et al. (2019) · Molecular Cell · doi:10.1016/j.molcel.2019.09.025 · Cited by 126 · see citing works
The NIH Somatic Cell Genome Editing program
Saha, K., Sontheimer, E. J., Brooks, P. J., Dwinell, M. R., Gersbach, C. A., Liu, D. R., Murray, S. A., Tsai, S. Q., Wilson, R. C., Anderson, D. G., et al. (2021) · Nature · doi:10.1038/s41586-021-03191-1 · Cited by 125 · see citing works
A Cas9 with PAM recognition for adenine dinucleotides
Chatterjee, P., Lee, J., Nip, L., Koseki, S., Tysinger, E., Sontheimer, E. J., Jacobson, J. M., & Jakimo, N. (2020) · Nature Communications · doi:10.1038/s41467-020-16117-8 · Cited by 121 · see citing works
CRISPR Modeling and Correction of Cardiovascular Disease
Liu, N., & Olson, E. N. (2022) · Circulation Research · doi:10.1161/circresaha.122.320496 · Cited by 118 · see citing works
Huang, T. P., Heins, Z. J., Miller, S. M., Wong, B. G., Balivada, P. A., Wang, T., Khalil, A. S., & Liu, D. R. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01410-2 · Cited by 115 · see citing works
Precise therapeutic gene correction by a simple nuclease-induced double-stranded break
Iyer, S., Suresh, S., Guo, D., Daman, K., Chen, J. C. J., Liu, P., Zieger, M. V., Luk, K., Roscoe, B. P., Mueller, C., et al. (2019) · Nature · doi:10.1038/s41586-019-1076-8 · Cited by 108 · see citing works
Ibraheim, R., Tai, P. W. L., Mir, A., Javeed, N., Wang, J., Rodríguez, T., Namkung, S., Nelson, S. J., Khokhar, E. S., Mintzer, E., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-26518-y · Cited by 109 · see citing works
Kong, X., Zhang, H., Li, G., Wang, Z., Kong, X., Wang, L., Xue, M., Zhang, W., Wang, Y., Lin, J., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-37829-7 · Cited by 106 · see citing works
Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins
Lee, J., Mou, H., Ibraheim, R., Liang, S.-Q., Liu, P., Xue, W., & Sontheimer, E. J. (2019) · RNA · doi:10.1261/rna.071704.119 · Cited by 100 · see citing works
Xiang, G., Li, Y., Sun, J., Huo, Y., Cao, S., Cao, Y., Guo, Y., Yang, L., Cai, Y., Zhang, Y. E., et al. (2023) · Nature Biotechnology · doi:10.1038/s41587-023-01857-x · Cited by 102 · see citing works
CRISPR/Cas System and Factors Affecting Its Precision and Efficiency
Javaid, N., & Choi, S. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.761709 · Cited by 98 · see citing works
In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice
Nahmad, A. D., Lazzarotto, C. R., Zelikson, N., Kustin, T., Tenuta, M., Huang, D., Reuveni, I., Nataf, D., Raviv, Y., Horovitz‐Fried, M., et al. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01328-9 · Cited by 94 · see citing works
Li, M., Yang, Y., Wang, Z., Zong, T., Fu, X., Aung, L. H. H., Wang, K., Wang, J., & Yu, T. (2020) · Angiogenesis · doi:10.1007/s10456-020-09750-w · Cited by 80 · see citing works
Rapid DNA unwinding accelerates genome editing by engineered CRISPR-Cas9
Eggers, A. R., Chen, K., Soczek, K. M., Tuck, O. T., Doherty, E. E., Xu, B., Trinidad, M. I., Thornton, B. W., Yoon, P. H., & Doudna, J. A. (2024) · Cell · doi:10.1016/j.cell.2024.04.031 · Cited by 72 · see citing works
Versatile and robust genome editing with Streptococcus thermophilus CRISPR1-Cas9Preprint
Agudelo, D., Carter, S., Velimirovic, M., Duringer, A., Rivest, J.-F., Levesque, S., Loehr, J., Mouchiroud, M., Cyr, D., Waters, P. J., et al. (2020) · Genome Research · doi:10.1101/gr.255414.119 · Cited by 74 · see citing works
Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector
Zhang, H., Bamidele, N., Liu, P., Ojelabi, O., Gao, X. D., Rodríguez, T., Cheng, H., Kelly, K. L., Watts, J. K., Xie, J., et al. (2022) · GEN Biotechnology · doi:10.1089/genbio.2022.0015 · Cited by 66 · see citing works
Li, Z., Wang, J., Xu, J.-P., Yang, X. F., & Yang, X. F. (2023) · Military Medical Research · doi:10.1186/s40779-023-00447-x · Cited by 68 · see citing works
The use of new CRISPR tools in cardiovascular research and medicine
Nishiga, M., Liu, C., Qi, L. S., & Wu, J. C. (2022) · Nature Reviews Cardiology · doi:10.1038/s41569-021-00669-3 · Cited by 67 · see citing works
Hu, Z., Zhang, C., Wang, S., Gao, S., Wei, J., Li, M., Hou, L., Mao, H., Wei, Y., Qi, T., et al. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab148 · Cited by 65 · see citing works
Advances in Genome Editing and Application to the Generation of Genetically Modified Rat Models
Chenouard, V., Remy, S., Tesson, L., Ménoret, S., Ouisse, L.-H., Cherifi, Y., & Anegon, I. (2021) · Frontiers in Genetics · doi:10.3389/fgene.2021.615491 · Cited by 57 · see citing works
Recent advances in therapeutic gene-editing technologies
Liu, D., Cao, D., & Han, R. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.026 · Cited by 62 · see citing works
Schmidt, M. J., Gupta, A. K., Bednarski, C., Gehrig-Giannini, S., Richter, F. M., Pitzler, C., Gamalinda, M., Galonska, C., Takeuchi, R., Wang, K., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-24454-5 · Cited by 57 · see citing works
PAM-flexible genome editing with an engineered chimeric Cas9
Zhao, L., Koseki, S. R. T., Silverstein, R. A., Amrani, N., Peng, C., Kramme, C., Savic, N., Pacesa, M., Rodríguez, T. C., Stan, T., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-41829-y · Cited by 59 · see citing works
Repurposing the Streptococcus mutans CRISPR-Cas9 System to Understand Essential Gene Function
Shields, R. C., Walker, A. R., Maricic, N., Chakraborty, B., Underhill, S. A. M., & Burne, R. A. (2020) · PLoS Pathogens · doi:10.1371/journal.ppat.1008344 · Cited by 52 · see citing works
Precise base editing with CC context-specificity using engineered human APOBEC3G-nCas9 fusions
Liu, Z., Chen, S., Shan, H., Jia, Y., Chen, M., Song, Y., Lai, L., & Li, Z. (2020) · BMC Biology · doi:10.1186/s12915-020-00849-6 · Cited by 53 · see citing works
Efficient Homology-Directed Repair with Circular Single-Stranded DNA Donors
Iyer, S., Mir, A., Vega‐Badillo, J., Roscoe, B. P., Ibraheim, R., Zhu, L. J., Lee, J., Liu, P., Luk, K., Mintzer, E., et al. (2022) · The CRISPR Journal · doi:10.1089/crispr.2022.0058 · Cited by 60 · see citing works
A highly specific CRISPR-Cas12j nuclease enables allele-specific genome editing
Wang, Y., Qi, T., Liu, J., Yang, Y., Wang, Z., Wang, Y., Wang, T., Li, M., Li, M., Lu, D., et al. (2023) · Science Advances · doi:10.1126/sciadv.abo6405 · Cited by 51 · see citing works
Synergistic engineering of CRISPR-Cas nucleases enables robust mammalian genome editing
Chen, Y., Hu, Y., Wang, X., Luo, S., Yang, N., Chen, Y., Li, Z., Zhou, Q., & Li, W. (2022) · The Innovation · doi:10.1016/j.xinn.2022.100264 · Cited by 48 · see citing works
Allemailem, K. S., Almatroodi, S. A., Almatroudi, A., Alrumaihi, F., Abdulmonem, W. A., Al‐Megrin, W. A. I., Aljamaan, A. N., Rahmani, A. H., & Khan, A. A. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24087052 · Cited by 51 · see citing works
Efficient plant genome engineering using a probiotic sourced CRISPR-Cas9 system
Zhong, Z., Liu, G., Tang, Z., Xiang, S., Yang, L., Huang, L., He, Y., Fan, T., Liu, S., Zheng, X., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-41802-9 · Cited by 54 · see citing works
Efficient base editing with high precision in rabbits using YFE-BE4max
Liu, Z., Chen, S., Shan, H., Jia, Y., Chen, M., Song, Y., Lai, L., & Li, Z. (2020) · Cell Death and Disease · doi:10.1038/s41419-020-2244-3 · Cited by 47 · see citing works
Highly efficient base editing with expanded targeting scope using SpCas9‐NG in rabbits
Liu, Z., Shan, H., Chen, S., Chen, M., Song, Y., Lai, L., & Li, Z. (2019) · The FASEB Journal · doi:10.1096/fj.201901587r · Cited by 46 · see citing works
Continuous directed evolution of a compact CjCas9 variant with broad PAM compatibility
Schmidheini, L., Mathis, N., Marquart, K. F., Rothgangl, T., Kissling, L., Böck, D., Chanez, C., Wang, J. P., Jínek, M., & Schwank, G. (2023) · Nature Chemical Biology · doi:10.1038/s41589-023-01427-x · Cited by 47 · see citing works
Wang, K., Gong, Q., & Ye, X. (2019) · Theoretical and Applied Genetics · doi:10.1007/s00122-019-03464-4 · Cited by 48 · see citing works
Tariq, A., Mushtaq, M., Yaqoob, H., Bhat, B. A., Zargar, S. M., Raza, A., Ali, S., Charagh, S., Mubarik, M. S., Zaman, Q. U., et al. (2023) · GM Crops & Food · doi:10.1080/21645698.2023.2219111 · Cited by 45 · see citing works
Emerging applications of gene editing technologies for the development of climate-resilient crops
Chavhan, R. L., Jaybhaye, S. G., Hinge, V. R., Deshmukh, A. S., Shaikh, U. S., Jadhav, P. K., Kadam, U. S., & Hong, J. C. (2025) · Frontiers in Genome Editing · doi:10.3389/fgeed.2025.1524767 · Cited by 43 · see citing works
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies
Yin, S., Zhang, M., Liu, Y., Sun, X., Guan, Y., Chen, X., Yang, L., Huo, Y., Yang, J., Zhang, X., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.11.014 · Cited by 44 · see citing works
Efficient and high-fidelity base editor with expanded PAM compatibility for cytidine dinucleotide
Liu, Z., Chen, S., Jia, Y., Shan, H., Chen, M., Song, Y., Lai, L., & Li, Z. (2021) · Science China Life Sciences · doi:10.1007/s11427-020-1775-2 · Cited by 43 · see citing works
Gene therapy for monogenic disorders: challenges, strategies, and perspectives
Zhang, Y., & Wu, Z. (2023) · Journal of genetics and genomics/Journal of Genetics and Genomics · doi:10.1016/j.jgg.2023.08.001 · Cited by 43 · see citing works
Therapeutic genome editing in cardiovascular diseases
Nishiga, M., Qi, L. S., & Wu, J. C. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.02.003 · Cited by 41 · see citing works
Alariqi, M., Ramadan, M., Yu, L., Hui, F., Hussain, A., Zhou, X., Yu, Y., Zhang, X., & Jin, S. (2025) · Advanced Science · doi:10.1002/advs.202416331 · Cited by 42 · see citing works
Wide Horizons of CRISPR-Cas-Derived Technologies for Basic Biology, Agriculture, and Medicine
Molla, K. A., Karmakar, S., & Islam, T. (2020) · Springer protocols handbooks/Springer protocols · doi:10.1007/978-1-0716-0616-2_1 · Cited by 41 · see citing works
A most formidable arsenal: genetic technologies for building a better mousePreprint
Clark, J. F., Dinsmore, C. J., & Soriano, P. M. (2020) · Genes & Development · doi:10.1101/gad.342089.120 · Cited by 40 · see citing works
Allele-specific genome targeting in the development of precision medicine
Wu, J., Tang, B., & Tang, Y. (2020) · Theranostics · doi:10.7150/thno.43298 · Cited by 38 · see citing works
Closely related type II-C Cas9 orthologs recognize diverse PAMs
Wei, J., Hou, L., Liu, J., Wang, Z., Gao, S., Qi, T., Gao, S., Sun, S., & Wang, Y. (2022) · eLife · doi:10.7554/elife.77825 · Cited by 35 · see citing works
CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing
Dasgupta, I., Flotte, T. R., & Keeler, A. M. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.013 · Cited by 37 · see citing works
The ScCas9++ variant expands the CRISPR toolbox for genome editing in plants
Liu, T., Zeng, D., Zheng, Z., Lin, Z., Xue, Y., Li, T., Xie, X., Ma, G., Liu, Y., & Zhu, Q. (2021) · Journal of Integrative Plant Biology · doi:10.1111/jipb.13164 · Cited by 35 · see citing works
Using CRISPR/Cas9 to model human liver disease
Alves‐Bezerra, M., Furey, N., Johnson, C. G., & Bissig, K. (2019) · JHEP Reports · doi:10.1016/j.jhepr.2019.09.002 · Cited by 32 · see citing works
Stojković, M., Han, D., Jeong, M., Stojković, P., & Stanković, K. M. (2021) · Stem Cells · doi:10.1002/stem.3353 · Cited by 34 · see citing works
Diverse Mechanisms of CRISPR-Cas9 Inhibition by Type II Anti-CRISPR Proteins
Hwang, S., & Maxwell, K. L. (2023) · Journal of Molecular Biology · doi:10.1016/j.jmb.2023.168041 · Cited by 33 · see citing works
Anti‐CRISPR proteins targeting the CRISPR‐Cas system enrich the toolkit for genetic engineering
Liu, Q., Zhang, H., & Huang, X. (2019) · FEBS Journal · doi:10.1111/febs.15139 · Cited by 32 · see citing works
Chen, G., Wei, T., Yang, H., Li, G., & Li, H. (2022) · Cells · doi:10.3390/cells11192964 · Cited by 32 · see citing works
Recent Advances in the Production of Genome-Edited Rats
Sato, M., Nakamura, S., Inada, E., & Takabayashi, S. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23052548 · Cited by 30 · see citing works
A new family of CRISPR‐type V nucleases with C‐rich PAM recognition
Urbaitis, T., Gasiūnas, G., Young, J. K., Hou, Z., Paulraj, S., Godliauskaitė, E., Juskeviciene, M. M., Stitilyte, M., Jasnauskaitė, M., Mabuchi, M. Y., et al. (2022) · EMBO Reports · doi:10.15252/embr.202255481 · Cited by 31 · see citing works
Breaking genetic shackles: The advance of base editing in genetic disorder treatment
Xu, F., Zheng, C., Xu, W., Zhang, S., Liu, S., Chen, X., & Yao, K. (2024) · Frontiers in Pharmacology · doi:10.3389/fphar.2024.1364135 · Cited by 30 · see citing works
CRISPR Tools for Engineering Prokaryotic Systems: Recent Advances and New Applications
Burbano, D. A., Kiattisewee, C., Karanjia, A. V., Cardiff, R. A. L., Faulkner, I., Sugianto, W., & Carothers, J. M. (2024) · Annual Review of Chemical and Biomolecular Engineering · doi:10.1146/annurev-chembioeng-100522-114706 · Cited by 32 · see citing works
Compact Cje3Cas9 for Efficient In Vivo Genome Editing and Adenine Base Editing
Chen, S., Liu, Z., Xie, W., Yu, H., Lai, L., & Li, Z. (2022) · The CRISPR Journal · doi:10.1089/crispr.2021.0143 · Cited by 30 · see citing works
Genome editing with type II‐C CRISPR‐Cas9 systems from Neisseria meningitidis in rice
Xu, R., Qin, R., Xie, H., Li, J., Liu, X., Zhu, M., Sun, Y., Yu, Y., Lu, P., & Wei, P. (2021) · Plant Biotechnology Journal · doi:10.1111/pbi.13716 · Cited by 28 · see citing works
Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9
Liu, Z., Chen, S., Xie, W., Song, Y., Li, J., Lai, L., & Li, Z. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.06.013 · Cited by 28 · see citing works
Compact SchCas9 Recognizes the Simple NNGR PAM
Wang, S., Mao, H., Hou, L., Hu, Z., Wang, Y., Qi, T., Tao, C., Yang, Y., Zhang, C., Li, M., et al. (2021) · Advanced Science · doi:10.1002/advs.202104789 · Cited by 23 · see citing works
CRISPR/Cas therapeutic strategies for autosomal dominant disorders
Caruso, S. M., Quinn, P. M. J., da Costa, B. L., & Tsang, S. H. (2022) · Journal of Clinical Investigation · doi:10.1172/jci158287 · Cited by 26 · see citing works
Recent advances in therapeutic CRISPR-Cas9 genome editing: mechanisms and applications
Zhou, L., & Yao, S. (2023) · Molecular Biomedicine · doi:10.1186/s43556-023-00115-5 · Cited by 27 · see citing works
Zhou, F., Yu, X., Gan, R., Ren, K., Chen, C., Ren, C., Cui, M., Liu, Y., Gao, Y., Wang, S., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad425 · Cited by 25 · see citing works
Ren, J., Hai, T., Chen, Y., Sun, K., Han, Z., Wang, J., Li, C., Wang, Q., Wang, L., Zhu, H., et al. (2023) · Science China Life Sciences · doi:10.1007/s11427-023-2407-0 · Cited by 24 · see citing works
AAV diffuses across zona pellucida for effortless gene delivery to fertilized eggs
Romeo, C., Chen, S., Goulding, E. H., Gorder, L. V., Schwartz, M., Walker, M., Scott, G., Scappini, E., Ray, M. K., & Martin, N. P. (2020) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2020.03.026 · Cited by 23 · see citing works
PpCas9 from Pasteurella pneumotropica — a compact Type II-C Cas9 ortholog active in human cells
Fedorova, I., Vasileva, A., Selkova, P., Abramova, M., Arseniev, A., Pobegalov, G. E., Kazalov, M., Musharova, O. S., Goryanin, I. I., Artamonova, D., et al. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa998 · Cited by 23 · see citing works
Wen, J., Cao, T., Wu, J., Chen, Y., Zhi, S., Huang, Y., Zhen, P., Wu, G., Aagaard, L., Zhong, J., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.05.010 · Cited by 23 · see citing works
Increasing the Targeting Scope of CRISPR Base Editing System Beyond NGG
Yu, S.-Y., Birkenshaw, A., Thomson, T., Carlaw, T., Zhang, L., & Ross, C. J. (2022) · The CRISPR Journal · doi:10.1089/crispr.2021.0109 · Cited by 23 · see citing works
CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing
Pedrazzoli, E., Demozzi, M., Visentin, E., Ciciani, M., Bonuzzi, I., Pezzè, L., Lucchetta, L., Maule, G., Amistadi, S., Esposito, F., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-47800-9 · Cited by 23 · see citing works
Takasugi, P., Wang, S., Truong, K. T., Drage, E. P., Kanishka, S. N., Higbee, M. A., Bamidele, N., Ojelabi, O., Sontheimer, E. J., & Gagnon, J. A. (2021) · Genetics · doi:10.1093/genetics/iyab196 · Cited by 21 · see citing works
Zhou, W., Yang, J., Zhang, Y., Hu, X., & Wang, W. (2022) · MedComm · doi:10.1002/mco2.155 · Cited by 22 · see citing works
A multifunctional Wnt regulator underlies the evolution of rodent stripe patterns
Johnson, M. R., Li, S., Guerrero‐Juarez, C. F., Miller, P. W., Brack, B. J., Mereby, S. A., Moreno, J. A., Feigin, C. Y., Gaska, J. M., Rivera‐Pérez, J. A., et al. (2023) · Nature Ecology & Evolution · doi:10.1038/s41559-023-02213-7 · Cited by 22 · see citing works
Yue, J.-J., Hong, C., Wei, P., Tsai, Y., & Lin, C. (2020) · Rice · doi:10.1186/s12284-019-0354-2 · Cited by 22 · see citing works
Computational design of anti-CRISPR proteins with improved inhibition potency
Mathony, J., Harteveld, Z., Schmelas, C., zu Belzen, J. U., Aschenbrenner, S., Sun, W. Y., Hoffmann, M. D., Stengl, C., Scheck, A., Georgeon, S., et al. (2020) · Nature Chemical Biology · doi:10.1038/s41589-020-0518-9 · Cited by 21 · see citing works
Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope
Bamidele, N., Zhang, H., Dong, X., Cheng, H., Gaston, N., Feinzig, H., Cao, H., Kelly, K. L., Watts, J. K., Xie, J., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-45763-5 · Cited by 22 · see citing works
Mechanistic insights into the versatile class II CRISPR toolbox
Zhang, F., & Huang, Z. (2021) · Trends in Biochemical Sciences · doi:10.1016/j.tibs.2021.11.007 · Cited by 19 · see citing works
- Authors
- Edraki, A., Mir, A., Ibraheim, R., Gainetdinov, I., Yoon, Y., Song, C.-Q., Cao, Y., Gallant, J., Xue, W., Rivera-Pérez, J. A., & Sontheimer, E. J.
- Affiliations
- University of Massachusetts Chan Medical School (US)
- Corresponding author
- Sontheimer, Erik J.
- ORCID
- Venue
- Molecular Cell
- Publisher
- Elsevier BV
- Published
- 2019-02
- Volume
- 73
- Issue
- 4
- Pages
- 714-726.e4
- ISSN
- 1097-2765 (ISSN-L)
- PMC
- PMC6386616
- OpenAlex
- W2906461396
- Cited by
- 265
- References
- 80
- Citations by year
- 2026: 27 · 2025: 27 · 2024: 36 · 2023: 56 · 2022: 41 · 2021: 26 · 2020: 35 · 2019: 15 · 2018: 1
- FWCI
- 6.81
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Molecular Cell (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- The Journal of the American Medical Association (JAMA) Network (American Medical Association) (submittedVersion)
- Funders
- National Institutes of Health (DP2 HL137167, P01 HL131471, R01 GM115911, R01 GM125797)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- Cas9, Neisseria meningitidis, CRISPR/Cas9, in vivo gene editing, adeno-associated virus delivery, PCSK9, hypercholesterolemia, off-target editing, protospacer adjacent motif
- MeSH terms
- Proprotein Convertase 9, Gene Editing, CRISPR-Associated Protein 9, RNA, Guide, CRISPR-Cas Systems, Dependovirus, Animals, DNA, Embryo Transfer, Female, Genetic Vectors, Humans, Liver, Mice, Inbred C57BL, Neisseria meningitidis, Substrate Specificity, Zygote, K562 Cells, HEK293 Cells, Nucleotide Motifs, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Cas Systems
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