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Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
Open accessAbstract
A PAMless base editor: CRISPR-Cas DNA base editing typically requires a specific motif for targeting known as a protospacer-adjacent motif (PAM). This requirement limits the sequences within a genome that can be targeted. Walton et al. engineered specific variants of the Streptococcus pyogenes Cas9 enzyme named SpG and SpRY that could recognize and edit a wider array of PAMs. Using SpRY, the authors were able to correct previously uneditable mutations associated with human disease. Although off-target effects were observed for these engineered Cas enzymes at levels similar to those of the wild-type enzyme, depending on the context, these engineered enzymes widen the potential applications of precision genome editing. Science , this issue p. 290
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“Efforts are ongoing to develop PAM-free Cas9 molecules to eliminate PAM site constraints altogether, thereby increasing the flexibility and effectiveness of genome editingthrough structure-based rational engineering or by altering amino acid substitutions in the PAM-interacting domain (Walton et al., 2020).”
From Emerging applications of gene editing technologies for the development of climate-resilient crops (2025) · cites this paper as Walton et al., 2020
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“CRISPR-Cas dominates the field because of the ease of using guide RNAs directing the CRISPR complex to specific DNA sequences and the increasing ability to target most sites in the genome.121,122”
From Fetal hemoglobin in sickle cell anemia (2020) · cites this paper as [122]
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“Building on this, Walton et al. (2020) introduced the SpG variant, which can recognize a broader range of NGN PAMs.”
From Emerging applications of gene editing technologies for the development of climate-resilient crops (2025) · cites this paper as Walton et al., 2020
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“These can be targeted in combination with evolved Cas9 variants such as SpCas9-NG20 or SpG.19”
From Protocol to create isogenic disease models from adult stem cell-derived organoids using next-generation CRISPR tools (2024) · cites this paper as [19]
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“These can be targeted with SpRY.19”
From Protocol to create isogenic disease models from adult stem cell-derived organoids using next-generation CRISPR tools (2024) · cites this paper as [19]
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“Cas9 genomic localization is delimited by the protospacer adjacent motif (PAM), which is NGG for SpCas9 while evolved variants of SpCas9 tolerate a broader range of PAM sequences17–21.”
From One-step generation of tumor models by base editor multiplexing in adult stem cell-derived organoids (2023) · cites this paper as [21]
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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
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A., & Liu, D. R. (2016) · Nature · doi:10.1038/nature17946 · Cited by 5,437
Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
Zetsche, B., Gootenberg, J. S., Abudayyeh, O. O., Slaymaker, I. M., Makarova, K. S., Essletzbichler, P., Volz, S. E., Joung, J., van der Oost, J., Regev, A., et al. (2015) · Cell · doi:10.1016/j.cell.2015.09.038 · Cited by 4,761
Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
Gaudelli, N. M., Komor, A. C., Rees, H. A., Packer, M. S., Badran, A. H., Bryson, D. I., & Liu, D. R. (2017) · Nature · doi:10.1038/nature24644 · Cited by 4,184
High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects
Kleinstiver, B. P., Pattanayak, V., Prew, M. S., Tsai, S. Q., Nguyen, N. T., Zheng, Z., & Joung, J. K. (2016) · Nature · doi:10.1038/nature16526 · Cited by 2,673
Self-citationRNA-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
Rationally engineered Cas9 nucleases with improved specificity
Slaymaker, I. M., Gao, L., Zetsche, B., Scott, D. A., Yan, W. X., & Zhang, F. (2016) · Science · doi:10.1126/science.aad5227 · Cited by 2,414
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
DNA interrogation by the CRISPR RNA-guided endonuclease Cas9
Sternberg, S. H., Redding, S., Jinek, M., Greene, E. C., & Doudna, J. A. (2014) · Nature · doi:10.1038/nature13011 · Cited by 1,822
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
Flagged first, then by citations
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Wang, J. Y., & Doudna, J. A. (2023) · Science · doi:10.1126/science.add8643 · Cited by 1,105
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 824
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Chen, P. J., & Liu, D. R. (2022) · Nature Reviews Genetics · doi:10.1038/s41576-022-00541-1 · Cited by 644
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
CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future
Uddin, F. Z., Rudin, C. M., & Sen, T. (2020) · Frontiers in Oncology · doi:10.3389/fonc.2020.01387 · Cited by 641
CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells
Kurt, I. C., Zhou, R., Iyer, S., Garcia, S. P., Miller, B. R., Langner, L. M., Grünewald, J., & Joung, J. K. (2021) · Nature Biotechnology · doi:10.1038/s41587-020-0609-x · Cited by 573
CRISPR-Cas9 DNA Base-Editing and Prime-Editing
Kantor, A., McClements, M., & MacLaren, R. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21176240 · Cited by 462
Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
Wang, S.-W., Gao, C., Zheng, Y.-M., Yi, L., Lu, J.-C., Huang, X.-Y., Cai, J.-B., Zhang, P.-F., Cui, Y.-H., & Ke, A.-W. (2022) · Molecular Cancer · doi:10.1186/s12943-022-01518-8 · Cited by 478
Base editing: advances and therapeutic opportunities
Porto, E. M., Komor, A. C., Slaymaker, I. M., & Yeo, G. W.-M. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0084-6 · Cited by 499
Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing
Naeem, M., Majeed, S., Hoque, M. Z., & Ahmad, I. (2020) · Cells · doi:10.3390/cells9071608 · Cited by 457
Engineering the next generation of cell-based therapeutics
Bashor, C. J., Hilton, I. B., Bandukwala, H. S., Smith, D. M., & Omid Veiseh, A. (2022) · Nature Reviews Drug Discovery · doi:10.1038/s41573-022-00476-6 · Cited by 472
CRISPR in cancer biology and therapy
Katti, A., Diaz, B. J., Caragine, C. M., Sanjana, N. E., & Dow, L. E. (2022) · Nature reviews. Cancer · doi:10.1038/s41568-022-00441-w · Cited by 457
PnB Designer: a web application to design prime and base editor guide RNAs for animals and plants
Siegner, S. M., Karasu, M. E., Schröder, M., Kontarakis, Z., & Corn, J. E. (2021) · BMC Bioinformatics · doi:10.1186/s12859-021-04034-6 · Cited by 436
Xu, Y., & Li, Z. (2020) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2020.08.031 · Cited by 457
Precise plant genome editing using base editors and prime editors
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Lin, Q., Jin, S., Zong, Y., Yu, H., Zhu, Z., Liu, G., Kou, L., Wang, Y., Qiu, J.-L., Li, J., et al. (2021) · Nature Biotechnology · doi:10.1038/s41587-021-00868-w · Cited by 325
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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 304
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Liu, P., Liang, S.-Q., Zheng, C., Mintzer, E., Zhao, Y. G., Ponnienselvan, K., Mir, A., Sontheimer, E. J., Gao, G., Flotte, T. R., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-22295-w · Cited by 273
CRISPR/cas systems redefine nucleic acid detection: Principles and methods
Wang, M., Zhang, R., & Li, J. (2020) · Biosensors and Bioelectronics · doi:10.1016/j.bios.2020.112430 · Cited by 273
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Zhang, H., Qin, C., An, C., Zheng, X., Wen, S., Chen, W., Liu, X., Lv, Z., Yang, P., Xu, W., et al. (2021) · Molecular Cancer · doi:10.1186/s12943-021-01431-6 · Cited by 264
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Discovery of target genes and pathways at GWAS loci by pooled single-cell CRISPR screens
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Xu, L. M., Zhang, C., Bailin, P. L., Wang, P., Gao, Y., Mohler, P. J., Mokadam, N. A., Ma, J., Arnold, W. D., & Han, R. (2021) · Nature Communications · doi:10.1038/s41467-021-23996-y · Cited by 118
Wang, Y., Cheng, H., Liu, Y., Liu, Y., Xiao, W., Zhang, K., Ni, X., Gao, N., Fan, L., Zhang, Z., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-21003-y · Cited by 108
Engineered prime editors with PAM flexibility
Kweon, J., Yoon, J.-K., Jang, A.-H., Shin, H. R., See, J.-E., Jang, G., Kim, J., & Kim, Y. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.02.022 · Cited by 111
Comparison of the Feasibility, Efficiency, and Safety of Genome Editing Technologies
Castro, N. G. G., Bjelic, J., Malhotra, G., Huang, C., & Alsaffar, S. H. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms221910355 · Cited by 110
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges
Kalter, N., Fuster‐García, C., Silva, A., Ronco-Díaz, V., Roncelli, S., Turchiano, G., Gorodkin, J., Cathomen, T., Benabdellah, K., Lee, C. M., et al. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102636 · Cited by 121
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 110
A review on CRISPR/Cas-based epigenetic regulation in plants
Jogam, P., Sandhya, D., Alok, A., Peddaboina, V., Allini, V. R., & Zhang, B. (2022) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2022.08.182 · Cited by 105
A versatile genetic engineering toolkit for E. coli based on CRISPR-prime editing
Tong, Y., Jørgensen, T. S., Whitford, C. M., Weber, T., & Lee, S. Y. (2021) · Nature Communications · doi:10.1038/s41467-021-25541-3 · Cited by 104
CRISPR-based gene editing technology and its application in microbial engineering
Wei, J., & Li, Y. (2023) · Engineering Microbiology · doi:10.1016/j.engmic.2023.100101 · Cited by 106
CRISPR engineering in organoids for gene repair and disease modelling
Geurts, M. H., & Clevers, H. (2023) · Nature Reviews Bioengineering · doi:10.1038/s44222-022-00013-5 · Cited by 103
Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations
Wang, L., Xue, W., Zhang, H., Gao, R., Qiu, H.-Y., Wei, J., Zhou, L., Lei, Y.-N., Wu, X., Li, X., et al. (2021) · Nature Cell Biology · doi:10.1038/s41556-021-00671-4 · Cited by 106
CRISPR screens in plants: approaches, guidelines, and future prospects
Gaillochet, C., Develtere, W., & Jacobs, T. B. (2021) · The Plant Cell · doi:10.1093/plcell/koab099 · Cited by 102
A design optimized prime editor with expanded scope and capability in plants
Xu, W., Yang, Y., Yang, B., Krueger, C. J., Xiao, Q., Zhao, S., Zhang, L., Kang, G., Wang, F., Yi, H., et al. (2021) · Nature Plants · doi:10.1038/s41477-021-01043-4 · Cited by 101
Green Revolution to Gene Revolution: Technological Advances in Agriculture to Feed the World
Hamdan, M. F., Noor, S. N. M., Abd-Aziz, N., Pua, T., & Tan, B. C. (2022) · Plants · doi:10.3390/plants11101297 · Cited by 107
Genetic glycoengineering in mammalian cells
Narimatsu, Y., Büll, C., Chen, Y., Wandall, H. H., Yang, Z., & Clausen, H. (2021) · Journal of Biological Chemistry · doi:10.1016/j.jbc.2021.100448 · Cited by 103
CRISPR-Based Genome Editing Tools: Insights into Technological Breakthroughs and Future Challenges
Mushtaq, M., Dar, A. A., Skalický, M., Tyagi, A., Bhagat, N., Basu, U., Bhat, B. A., Zaid, A., Ali, S., Dar, T. U. H., et al. (2021) · Genes · doi:10.3390/genes12060797 · Cited by 100
Genome editing mediated by SpCas9 variants with broad non-canonical PAM compatibility in plants
Li, J., Xu, R., Qin, R., Liu, X., Kong, F., & Wei, P. (2020) · Molecular Plant · doi:10.1016/j.molp.2020.12.017 · Cited by 100
Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing
Maule, G., Arosio, D., & Cereseto, A. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21113903 · Cited by 102
Advances in the profiling of N6-methyladenosine (m6A) modifications
Zheng, H., Zhang, X., & Sui, N. (2020) · Biotechnology Advances · doi:10.1016/j.biotechadv.2020.107656 · Cited by 98
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 106
Base-editing mutagenesis maps alleles to tune human T cell functions
Schmidt, R. L. J., Ward, C. C., Dajani, R., Armour-Garb, Z., Ota, M., Allain, V., Hernandez, R., Layeghi, M., Xing, G., Goudy, L., et al. (2023) · Nature · doi:10.1038/s41586-023-06835-6 · Cited by 101
DNA interference states of the hypercompact CRISPR–CasΦ effector
Pausch, P., Soczek, K. M., Herbst, D. A., Tsuchida, C. A., Al-Shayeb, B., Banfield, J. F., Nogales, E., & Doudna, J. A. (2021) · Nature Structural & Molecular Biology · doi:10.1038/s41594-021-00632-3 · Cited by 101
CRISPR-Cas9 System for Plant Genome Editing: Current Approaches and Emerging Developments
Montecillo, J. A. V., Chu, L. L., & Bae, H. (2020) · Agronomy · doi:10.3390/agronomy10071033 · Cited by 99
Plant base editing and prime editing: The current status and future perspectives
Li, J., Zhang, C., He, Y., Li, S., Yan, L., Li, Y., Zhu, Z., & Xia, L. (2022) · Journal of Integrative Plant Biology · doi:10.1111/jipb.13425 · Cited by 94
Zhu, Y. (2022) · BioMed Research International · doi:10.1155/2022/9978571 · Cited by 103
Metadata
- Authors
- Walton, R. T., Christie, K. A., Whittaker, M. N., & Kleinstiver, B. P.
- Affiliations
- Massachusetts General Hospital (US)
- Harvard University (US)
- Corresponding author
- Kleinstiver, Benjamin P.
- Venue
- Science
- Published
- 2020-04-17
- Volume
- 368
- Issue
- 6488
- Pages
- 290-296
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- PMC
- PMC7297043
- OpenAlex
- W3013504322
- Semantic Scholar
- 84609b61a82a11e3122a81b5bdae20a2822e577b
- Cited by
- 1,233
- References
- 48
- Citations by year
- 2026: 157 · 2025: 200 · 2024: 190 · 2023: 213 · 2022: 203 · 2021: 177 · 2020: 93
- FWCI
- 56.07
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Heart, Lung, and Blood Institute (P01-HL142494)
- National Cancer Institute (R00-CA218870)
- American Society of Gene and Cell Therapy
- Margaret Q. Landenberger Research Foundation
- 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
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Keywords
- CRISPR/Cas9, SpCas9, SPG, SpRY, base editing, genome editing, human cells
- MeSH terms
- Protein Domains, Gene Editing, CRISPR-Associated Protein 9, Humans, Substrate Specificity, Mutagenesis, Gene Targeting, Genetic Predisposition to Disease, HEK293 Cells, CRISPR-Cas Systems
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