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RNA-Guided Human Genome Engineering via Cas9
Open accessAbstract
Genome Editing: Clustered regularly interspaced short palindromic repeats (CRISPR) function as part of an adaptive immune system in a range of prokaryotes: Invading phage and plasmid DNA is targeted for cleavage by complementary CRISPR RNAs (crRNAs) bound to a CRISPR-associated endonuclease (see the Perspective by van der Oost ). Cong et al. (p. 819 , published online 3 January) and Mali et al. (p. 823 , published online 3 January) adapted this defense system to function as a genome editing tool in eukaryotic cells.
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“Upon target recognition, the DNA strands are opened in an R-loop and individually cleaved, which results in a double-strand break (DSB)22,23”
From One-step generation of tumor models by base editor multiplexing in adult stem cell-derived organoids (2023) · cites this paper as [23]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“The technique also allows DNA sequence changes in pluripotent embryonic stem cells (4) that can then be cultured to produce specific tissues, such as cardiomyocytes or neurons (5).”
From A prudent path forward for genomic engineering and germline gene modification (2015) · cites this paper as [4]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“In January 2013, almost simultaneously, five research articles authored by different research teams appeared, all reporting that they had achieved the goal.”
From CRISPR–Cas9: A History of Its Discovery and Ethical Considerations of Its Use in Genome Editing (2022) · cites this paper as [34]
Experimental — accuracy not yet measuredThe cited source says what the claim attributes to it.
In the citing paper
“Mali et al. created a novel version of the genome-editing tool applicable to mammalian cells, termed the CRISPR/Cas9 system, which is based on modifications of the Streptococcus pyogenes type II CRISPR system in crRNA fused to trans-encoded tracrRNA 13 .”
From Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus (2013) · cites this paper as [13]
The cited source says what the claim attributes to it.
In the citing paper
“The pigs were engineered to carry 69 genomic edits, using the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (Cas9)-mediated nonhomologous end joining and homology-directed repair30,31, and recombinase-mediated cassette exchange32 (Fig. 2a, Extended Data Fig. 1a and Supplementary Table 2).”
From Design and testing of a humanized porcine donor for xenotransplantation (2023) · cites this paper as [31]
In the citing paper
“When a double-stranded break is introduced into genomic DNA, cells can repair the break by using a DNA molecule with high sequence similarity as a template. If an artificial template is provided while the DNA break is introduced, this pathway provides a mean to incorporate desired DNA sequences into the genome34,35”
From CRISPR-based functional genomics for neurological disease (2020) · cites this paper as [35]
In the citing paper
“The discovery, made less than 10 years ago, that a simple two-component system of a Cas protein (Streptococcus pyogenes Cas9) bound to a single guide RNA (sgRNA) can function as a programmable nuclease33 that can be deployed to cleave specific sequences in mammalian genomes34,35 has been transformative for biomedical research and the development of gene therapies.”
From CRISPR-based functional genomics for neurological disease (2020) · cites this paper as [35]
In the citing paper
“Recently, the CRISPR/Cas system has been successfully applied to human cells19,20,21,22 and several model organisms for genome editing.23,24,25,26”
From The CRISPR/Cas9 system facilitates clearance of the intrahepatic HBV templates in vivo (2014) · cites this paper as [19]
In the citing paper
“By bioinformatic screening and efficiency/off-target prediction (14, 15), we identified four gRNA targets (protospacers; LTRs A-D) that avoid conserved transcription factor binding sites, minimizing the likelihood of altering host gene expression”
From RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection (2014) · cites this paper as [15]
In the citing paper
“Compared with well-known, site-specific genome editing technologies, such as ZFNs and TALENs, the new CRISPR-Cas approach is more convenient and efficient [10], [12].”
From High-efficiency targeted editing of large viral genomes by RNA-guided nucleases (2014) · cites this paper as [10]
In the citing paper
“Clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 is a naturally occurring bacterial immune system that uses a novel nuclease system to protect bacteria from phage infection (6-9), and it has recently been harnessed for a variety of genome-engineering applications (10)(11)(12)(13)(14)(15)(16)(17).”
From RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection (2014) · cites this paper as [13]
In the citing paper
“Moreover, no significant cytotoxicity was observed [87,88].”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [88]
In the citing paper
“although Cas9 protein naturally induces double stranded DNA breaks by cutting each strand with two separate motifs, a mutation within one of the enzymatic motifs of Cas9 converted it into a valuable nickase with the capability to induce homologous recombination”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [88]
In the citing paper
“and the mutant is much safer to use with less off-target effects [88].”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [88]
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Citations
Cites
BEDTools: a flexible suite of utilities for comparing genomic features
Quinlan, A. R., & Hall, I. M. (2010) · Bioinformatics · doi:10.1093/bioinformatics/btq033 · Cited by 27,437
Ultrafast and memory-efficient alignment of short DNA sequences to the human genome.
Langmead, B., Trapnell, C., Pop, M., & Salzberg, S. L. (2009) · Genome Biology · doi:10.1186/gb-2009-10-3-r25 · Cited by 21,849
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
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
The Human Genome Browser at UCSC
Kent, W. J., Sugnet, C. W., Furey, T. S., Roskin, K. M., Pringle, T. H., Zahler, A. M., & Haussler, A. D. (2002) · Genome Research · doi:10.1101/gr.229102 · Cited by 10,400
Lorenz, R., Bernhart, S. H., Höner zu Siederdissen, C., Tafer, H., Flamm, C., Stadler, P. F., & Hofacker, I. L. (2011) · Algorithms for Molecular Biology · doi:10.1186/1748-7188-6-26 · Cited by 5,098
A System for Stable Expression of Short Interfering RNAs in Mammalian Cells
Brummelkamp, T. R., Bernards, R., & Agami, R. (2002) · Science · doi:10.1126/science.1068999 · Cited by 4,295
Mathews, D. H., Sabina, J., Zuker, M., & Turner, D. H. (1999) · Journal of Molecular Biology · doi:10.1006/jmbi.1999.2700 · Cited by 3,795
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
The accessible chromatin landscape of the human genome
Thurman, R. E., Rynes, E., Humbert, R., Vierstra, J., Maurano, M. T., Haugen, E., Sheffield, N. C., Stergachis, A. B., Wang, H., Vernot, B., et al. (2012) · Nature · doi:10.1038/nature11232 · Cited by 2,679
Cited by
Sorted by citations
Items 1,001–1,100 of 1,104
CRISPR-based functional genomics for neurological disease
Kampmann, M. (2020) · Nature Reviews Neurology · doi:10.1038/s41582-020-0373-z · Cited by 163
Markerless chromosomal gene deletion in Clostridium beijerinckii using CRISPR/Cas9 system
Wang, Y., Zhang, Z.-T., Seo, S.-O., Choi, K., Lu, T., Jin, Y.-S., & Blaschek, H. P. (2015) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2015.02.005 · Cited by 161
Ppm1b negatively regulates necroptosis through dephosphorylating Rip3
Chen, W., Wu, J., Li, L., Zhang, Z., Ren, J., Liang, Y., Chen, F., Yang, C., Zhou, Z., Sean Su, S., et al. (2015) · Nature Cell Biology · doi:10.1038/ncb3120 · Cited by 161
Genomic Perspectives of Transcriptional Regulation in Forebrain Development
Nord, A. S., Pattabiraman, K., Visel, A., & Rubenstein, J. L. R. (2015) · Neuron · doi:10.1016/j.neuron.2014.11.011 · Cited by 152
The power of multiplexed functional analysis of genetic variants
Gasperini, M., Starita, L., & Shendure, J. (2016) · Nature Protocols · doi:10.1038/nprot.2016.135 · Cited by 160
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
Moreno, A. M., Fu, X., Zhu, J., Katrekar, D., Shih, Y.-R. V., Marlett, J., Cabotaje, J., Tat, J., Naughton, J., Lisowski, L., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.04.017 · Cited by 159
Self-citationTGFβ signaling directs serrated adenomas to the mesenchymal colorectal cancer subtype
Fessler, E., Drost, J., van Hooff, S. R., Linnekamp, J. F., Wang, X., Jansen, M., De Sousa E Melo, F., Prasetyanti, P. R., IJspeert, J. E., Franitza, M., et al. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201606184 · Cited by 159
Structural basis for the altered PAM specificities of engineered CRISPR-cas9
Hirano, S., Nishimasu, H., Ishitani, R., & Nureki, O. (2016) · Molecular Cell · doi:10.1016/j.molcel.2016.02.018 · Cited by 152
Current status and perspectives of genome editing technology for microalgae
Jeon, S., Lim, J.-M., Lee, H.-G., Shin, S.-E., Kang, N. K., Park, Y.-I., Oh, H.-M., Jeong, W.-J., Jeong, B.-R., & Chang, Y. K. (2017) · Biotechnology for Biofuels · doi:10.1186/s13068-017-0957-z · Cited by 154
Graphene oxide-mediated Cas9/sgRNA delivery for efficient genome editing
Yue, H., Zhou, X., Cheng, M., & Xing, D. (2018) · Nanoscale · doi:10.1039/c7nr07999k · Cited by 150
Ortinski, P. I., O’Donovan, B., Dong, X., & Kantor, B. (2017) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.04.002 · Cited by 146
High-efficiency targeted editing of large viral genomes by RNA-guided nucleases
Bi, Y., Sun, L., Gao, D., Ding, C., Li, Z., Li, Y., Cun, W., & Li, Q. (2014) · PLoS Pathogens · doi:10.1371/journal.ppat.1004090 · Cited by 148
Wang, Q., Liberti, M. V., Liu, P., Deng, X., Liu, Y., Locasale, J. W., & Lai, L. (2017) · Cell Chemical Biology · doi:10.1016/j.chembiol.2016.11.013 · Cited by 147
Kim, H. K., Lee, S., Kim, Y., Park, J., Min, S., Choi, J. W., Huang, T. P., Yoon, S., Liu, D. R., & Kim, H. H. (2020) · Nature Biomedical Engineering · doi:10.1038/s41551-019-0505-1 · Cited by 145
Stem cell models of Alzheimer’s disease: progress and challenges
Arber, C., Lovejoy, C., & Wray, S. (2017) · Alzheimer's Research & Therapy · doi:10.1186/s13195-017-0268-4 · Cited by 143
Genome editing assessment using CRISPR Genome Analyzer (CRISPR-GA)
Güell, M., Yang, L., & Church, G. M. (2014) · Bioinformatics · doi:10.1093/bioinformatics/btu427 · Cited by 142
Self-citationGonatopoulos-Pournatzis, T., Aregger, M., Brown, K. R., Farhangmehr, S., Braunschweig, U., Ward, H. N., Ha, K. C. H., Weiss, A., Billmann, M., Durbic, T., et al. (2020) · Nature Biotechnology · doi:10.1038/s41587-020-0437-z · Cited by 142
Adenoviral vector DNA for accurate genome editing with engineered nucleases
Holkers, M., Maggio, I., Henriques, S. F. D., Janssen, J. M., Cathomen, T., & Gonçalves, M. A. F. V. (2014) · Nature Methods · doi:10.1038/nmeth.3075 · Cited by 138
Hamilton, P. J., & Nestler, E. J. (2019) · Current Opinion in Neurobiology · doi:10.1016/j.conb.2019.05.005 · Cited by 137
RNA Interference (RNAi) Screening in Drosophila
Heigwer, F., Port, F., & Boutros, M. (2018) · Genetics · doi:10.1534/genetics.117.300077 · Cited by 125
Generation and validation of homozygous fluorescent knock-in cells using CRISPR-Cas9 genome editing
Koch, B., Nijmeijer, B., Kueblbeck, M., Cai, Y., Walther, N., & Ellenberg, J. (2018) · Nature Protocols · doi:10.1038/nprot.2018.042 · Cited by 134
A new class of temporarily phenotypic enhancers identified by CRISPR/Cas9-mediated genetic screening
Diao, Y., Li, B., Meng, Z., Jung, I., Lee, A. Y., Dixon, J., Maliskova, L., Guan, K.-L., Shen, Y., & Ren, B. (2016) · Genome Research · doi:10.1101/gr.197152.115 · Cited by 122
One-step generation of triple knockout CHO cell lines using CRISPR/Cas9 and fluorescent enrichment
Grav, L. M., Lee, J. S., Gerling, S., Kallehauge, T. B., Hansen, A. H., Kol, S., Lee, G. M., Pedersen, L. E., & Kildegaard, H. F. (2015) · Biotechnology Journal · doi:10.1002/biot.201500027 · Cited by 131
Gene therapy and genome surgery in the retina
DiCarlo, J. E., Mahajan, V. B., & Tsang, S. H. (2018) · Journal of Clinical Investigation · doi:10.1172/jci120429 · Cited by 127
Self-citationZhang, H., Xue, C., Shah, R., Bermingham, K., Hinkle, C. C., Li, W., Rodrigues, A., Tabita-Martinez, J., Millar, J. S., Cuchel, M., et al. (2015) · Circulation Research · doi:10.1161/circresaha.117.305860 · Cited by 126
Protospacer adjacent motif (PAM)-distal sequences engage CRISPR Cas9 DNA target cleavage
Cencic, R., Miura, H., Malina, A., Robert, F., Ethier, S., Schmeing, T. M., Dostie, J., & Pelletier, J. (2014) · PLoS ONE · doi:10.1371/journal.pone.0109213 · Cited by 117
Kluesner, M. G., Lahr, W. S., Lonetree, C.-L., Smeester, B. A., Qiu, X., Slipek, N. J., Claudio Vázquez, P. N., Pitzen, S. P., Pomeroy, E. J., Vignes, M. J., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-22009-2 · Cited by 121
DNA repair and aging: The impact of the p53 family
Nicolai, S., Rossi, A., Di Daniele, N., Melino, G., Annicchiarico-Petruzzelli, M., & Raschellà, G. (2015) · Aging · doi:10.18632/aging.100858 · Cited by 118
Lysosomal Signaling Licenses Embryonic Stem Cell Differentiation via Inactivation of Tfe3
Villegas, F., Lehalle, D., Mayer, D., Rittirsch, M., Stadler, M. B., Zinner, M., Olivieri, D., Vabres, P., Duplomb-Jego, L., De Bont, E. S. J. M., et al. (2019) · Cell Stem Cell · doi:10.1016/j.stem.2018.11.021 · Cited by 110
Simplified adenine base editors improve adenine base editing efficiency in rice
Hua, K., Tao, X., Liang, W., Zhang, Z., Gou, R., & Zhu, J. (2020) · Plant Biotechnology Journal · doi:10.1111/pbi.13244 · Cited by 108
DIG-Seq: A genome-wide CRISPR off-target profiling method using chromatin DNA
Kim, D., & Kim, J.-S. (2018) · Genome Research · doi:10.1101/gr.236620.118 · Cited by 108
Tools for efficient genome editing; ZFN, TALEN, and CRISPR
Shamshirgaran, Y., Liu, J., Sumer, H., Verma, P. J., & Taheri-Ghahfarokhi, A. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2301-5_2 · Cited by 114
Biodegradable Amino-Ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo
Zhang, X., Li, B., Luo, X., Zhao, W., Jiang, J., Zhang, C., Gao, M., Chen, X., & Dong, Y. (2017) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.7b08163 · Cited by 107
A Nuclear Export Signal Is Required for cGAS to Sense Cytosolic DNA
Sun, H., Huang, Y., Mei, S., Xu, F., Liu, X., Zhao, F., Yin, L., Zhang, D., Wei, L., Wu, C., et al. (2021) · Cell Reports · doi:10.1016/j.celrep.2020.108586 · Cited by 105
Molecular biology at the cutting edge: A review on CRISPR/CAS9 gene editing for undergraduates
Thurtle‐Schmidt, D. M., & Lo, T. (2018) · Biochemistry and Molecular Biology Education · doi:10.1002/bmb.21108 · Cited by 103
Genome Editing Weds CRISPR: What Is in It for Phytoremediation?
Basharat, Z., Novo, L. A. B., & Yasmin, A. (2018) · Plants · doi:10.3390/plants7030051 · Cited by 104
Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo
Park, H. M., Liu, H., Wu, J., Chong, A., Mackley, V., Fellmann, C., Rao, A., Jiang, F., Chu, H., Murthy, N., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-05641-3 · Cited by 102
Structural basis for the altered PAM recognition by engineered CRISPR-Cpf1
Nishimasu, H., Yamano, T., Gao, L., Zhang, F., Ishitani, R., & Nureki, O. (2017) · Molecular Cell · doi:10.1016/j.molcel.2017.04.019 · Cited by 102
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
Gaj, T., Staahl, B. T., Rodrigues, G. M. C., Limsirichai, P., Ekman, F. K., Doudna, J. A., & Schaffer, D. V. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx154 · Cited by 89
CRISPR-based bioengineering in microalgae for production of industrially important biomolecules
Dhokane, D., Shaikh, A., Yadav, A., Giri, N., Bandyopadhyay, A., Dasgupta, S., & Bhadra, B. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1267826 · Cited by 106
Targeting mutant KRAS with CRISPR-Cas9 controls tumor growth
Kim, W., Lee, S., Kim, H. S., Song, M., Cha, Y. H., Kim, Y.-H., Shin, J., Lee, E.-S., Joo, Y., Song, J. J., et al. (2018) · Genome Research · doi:10.1101/gr.223891.117 · Cited by 97
Xu, L., Zhao, L., Gao, Y., Xu, J., & Han, R. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw1048 · Cited by 91
Targeted Heritable Mutation and Gene Conversion by Cas9-CRISPR in Caenorhabditis elegans
Katic, I., & Großhans, H. (2013) · Genetics · doi:10.1534/genetics.113.155754 · Cited by 95
Antibody glycoengineering strategies in mammalian cells
Wang, Q., Chung, C., Chough, S., & Betenbaugh, M. J. (2018) · Biotechnology and Bioengineering · doi:10.1002/bit.26567 · Cited by 95
Synthetic modified messenger RNA for therapeutic applications
Gao, M., Zhang, Q., Feng, X.-H., & Liu, J. (2021) · Acta Biomaterialia · doi:10.1016/j.actbio.2021.06.020 · Cited by 89
Exosome/Liposome-like Nanoparticles: New Carriers for CRISPR Genome Editing in Plants
Alghuthaymi, M. A., Ahmad, A., Khan, Z., Khan, S. H., Ahmed, F. K., Faiz, S., Nepovimova, E., Kuča, K., & Abd-Elsalam, K. A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22147456 · Cited by 83
“The toolbox of CRISPR/Cas9 was adapted from bacteria as well as Archaea and includes engineered nucleases [93,94].”
MethodLiu, J., Song, T., Zhou, W., Xing, L., Wang, S., Ho, M., Peng, Z., Tai, Y.-T., Hideshima, T., Anderson, K. C., et al. (2019) · Leukemia · doi:10.1038/s41375-018-0205-y · Cited by 83
Kumar, N., Stanford, W., de Solis, C., Aradhana, Abraham, N. D., Dao, T.-M. J., Thaseen, S., Sairavi, A., Gonzalez, C. U., & Ploski, J. E. (2018) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2018.00413 · Cited by 79
Horie, K., Inoue, K., Suzuki, S., Adachi, S., Yada, S., Hirayama, T., Hidema, S., Young, L. J., & Nishimori, K. (2019) · Hormones and Behavior · doi:10.1016/j.yhbeh.2018.10.011 · Cited by 77
Newer Gene Editing Technologies toward HIV Gene Therapy
Manjunath, N., Yi, G., Dang, Y., & Shankar, P. (2013) · Viruses · doi:10.3390/v5112748 · Cited by 77
DNA Methylation and Its Implications and Accessibility for Neuropsychiatric Therapeutics
Day, J. J., Kennedy, A. J., & Sweatt, J. D. (2015) · Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-010814-124527 · Cited by 78
In vivo locus-specific editing of the neuroepigenome
Yim, Y. Y., Teague, C. D., & Nestler, E. J. (2020) · Nature Reviews Neuroscience · doi:10.1038/s41583-020-0334-y · Cited by 76
Gene editing for plant resistance to abiotic factors: a systematic review
Nascimento, F. D. S., Rocha, A. D. J., Soares, J. M. D. S., Mascarenhas, M. S., Ferreira, M. D. S., Morais Lino, L. S., Ramos, A. P. D. S., Diniz, L. E. C., Mendes, T. A. D. O., Ferreira, C. F., et al. (2023) · Plants · doi:10.3390/plants12020305 · Cited by 71
Liang, Y., Iqbal, Z., Wang, J., Xu, L., Xu, X., Ouyang, K., Zhang, H., Lu, J., Duan, L., & Xia, J. (2022) · Biomaterials Science · doi:10.1039/d2bm00480a · Cited by 72
Towards a new era in medicine: therapeutic genome editing
Porteus, M. H. (2015) · Genome Biology · doi:10.1186/s13059-015-0859-y · Cited by 69
Genetic engineering in organoids
Teriyapirom, I., Batista-Rocha, A. S., & Koo, B.-K. (2021) · Journal of Molecular Medicine · doi:10.1007/s00109-020-02029-z · Cited by 68
Genome Editing in iPSC-Based Neural Systems: From Disease Models to Future Therapeutic Strategies
McTague, A., Rossignoli, G., Ferrini, A., Barral, S., & Kurian, M. A. (2021) · Frontiers in Genome Editing · doi:10.3389/fgeed.2021.630600 · Cited by 67
One-step generation of tumor models by base editor multiplexing in adult stem cell-derived organoids
Geurts, M. H., Gandhi, S., Boretto, M. G., Akkerman, N., Derks, L. L. M., van Son, G., Celotti, M., Harshuk-Shabso, S., Peci, F., Begthel, H., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-40701-3 · Cited by 66
“Upon target recognition, the DNA strands are opened in an R-loop and individually cleaved, which results in a double-strand break (DSB) 22,23 Subsequent repair of the DSB by non-homologous end joining (NHEJ) mechanism can result in knock-out of the gene upon gain or loss of base pairs at the break…”
Tanihara, F., Hirata, M., Nguyen, N. T., Le, Q. A., Hirano, T., Takemoto, T., Nakai, M., Fuchimoto, D.-I., & Otoi, T. (2018) · PLOS ONE · doi:10.1371/journal.pone.0206360 · Cited by 63
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?
Wagner, D. L., Peter, L., & Schmueck-Henneresse, M. (2021) · Gene Therapy · doi:10.1038/s41434-021-00232-2 · Cited by 63
Customizing the genome as therapy for the β-hemoglobinopathies.
Canver, M. C., & Orkin, S. H. (2016) · Blood · doi:10.1182/blood-2016-01-678128 · Cited by 61
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
Self-citationQiang, L., Fujita, R., & Abeliovich, A. (2013) · Neuron · doi:10.1016/j.neuron.2013.06.002 · Cited by 59
Wu, Y., Ren, Q., Zhong, Z., Liu, G., Han, Y., Bao, Y., Liu, L., Xiang, S., Liu, S., Tang, X., et al. (2022) · Plant Biotechnology Journal · doi:10.1111/pbi.13838 · Cited by 56
Bannister, S., Antonova, O., Polo, A., Lohs, C., Hallay, N., Valinciute, A., Raible, F., & Tessmar-Raible, K. (2014) · Genetics · doi:10.1534/genetics.113.161091 · Cited by 56
“The genotyping and rapid larval digestion protocols that we introduced here can also be equally applied to other genome-editing systems, such as the recently described Cas9/CRISPR system (Cong et al. 2013; Mali et al. 2013).”
MethodVerbrugge, S. A. J., Schönfelder, M., Becker, L., Yaghoob Nezhad, F., Hrabě de Angelis, M., & Wackerhage, H. (2018) · Frontiers in Physiology · doi:10.3389/fphys.2018.00553 · Cited by 56
Wang, E., Kawaoka, S., Roe, J.-S., Shi, J., Hohmann, A. F., Xu, Y., Bhagwat, A. S., Suzuki, Y., Kinney, J. B., & Vakoc, C. R. (2015) · eLife · doi:10.7554/elife.06377 · Cited by 54
“To further evaluate whether the essential function of TRIM33 is due to its occupancy at the −117 region, we employed CRISPR-Cas9 to delete this element (Cong et al., 2013; Mali et al., 2013) (Figure 4C,D).”
MethodSelective targeting of KRAS oncogenic alleles by CRISPR/Cas9 inhibits proliferation of cancer cells
Lee, W., Lee, J. H., Jun, S., Lee, J. H., & Bang, D. (2018) · Scientific Reports · doi:10.1038/s41598-018-30205-2 · Cited by 53
Recent Progress and Challenges in CRISPR-Cas9 Engineered Algae and Cyanobacteria
Patel, V. K., Das, A., Kumari, R., & Kajla, S. (2023) · Algal Research · doi:10.1016/j.algal.2023.103068 · Cited by 50
Tran, N. T., Danner, E., Li, X., Graf, R., Lebedin, M., de la Rosa, K., Kühn, R., Rajewsky, K., & Chu, V. T. (2022) · Science Advances · doi:10.1126/sciadv.abm9106 · Cited by 49
Gao, Z., Harwig, A., Berkhout, B., & Herrera-Carrillo, E. (2017) · Transcription · doi:10.1080/21541264.2017.1322170 · Cited by 47
Kulcsár, P. I., Tálas, A., Tóth, E., Nyeste, A., Ligeti, Z., Welker, Z., & Welker, E. (2020) · Nature Communications · doi:10.1038/s41467-020-15021-5 · Cited by 45
Sheng, C., Mendler, I.-H., Rieke, S., Snyder, P., Jentsch, M., Friedrich, D., Drossel, B., & Loewer, A. (2019) · Cell Reports · doi:10.1016/j.celrep.2019.03.031 · Cited by 45
Horie, K., Inoue, K., Nishimori, K., & Young, L. J. (2020) · Neuroscience · doi:10.1016/j.neuroscience.2020.08.023 · Cited by 46
“Cas9 induces a double strand break into the target sequence and insertions or deletions are induced into the target sequence while it is repaired by endogenous DNA repair pathways (Cho et al., 2013; Cong et al., 2013; Mali et al., 2013; Wang et al., 2013).”
BackgroundNakaoka, H., Gurumurthy, A., Hayano, T., Ahmadloo, S., Omer, W. H., Yoshihara, K., Yamamoto, A., Kurose, K., Enomoto, T., Akira, S., et al. (2016) · PLOS Genetics · doi:10.1371/journal.pgen.1005893 · Cited by 46
Amann, T., Schmieder, V., Faustrup Kildegaard, H., Borth, N., & Andersen, M. R. (2019) · Biotechnology and Bioengineering · doi:10.1002/bit.27101 · Cited by 46
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
MEK inhibitors block growth of lung tumours with mutations in ataxia–telangiectasia mutated
Smida, M., Fece de la Cruz, F., Kerzendorfer, C., Uras, I. Z., Mair, B., Mazouzi, A., Suchankova, T., Konopka, T., Katz, A. M., Paz, K., et al. (2016) · Nature Communications · doi:10.1038/ncomms13701 · Cited by 44
“As complementaryDNA add-back experiments are complicated by the large size of ATM (350 kDa) and poor transfectability of the lung cancer cell lines, we next employed RNA-guided nucleases (RGNs) 22 as an alternative approach to inactivate ATM and confirm the synthetic lethal interaction.”
Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease
Kim, E. Y., Page, P., Dellefave-Castillo, L. M., McNally, E. M., & Wyatt, E. J. (2016) · Skeletal Muscle · doi:10.1186/s13395-016-0103-9 · Cited by 43
The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD
Wang, J.-Z., Wu, P., Shi, Z.-M., Xu, Y.-L., & Liu, Z.-J. (2017) · Brain and Development · doi:10.1016/j.braindev.2017.03.024 · Cited by 41
Human iPSC application in Alzheimer’s disease and tau-related neurodegenerative diseases
TCW, J. (2019) · Neuroscience Letters · doi:10.1016/j.neulet.2019.01.043 · Cited by 39
Zhan, H., Xie, H., Zhou, Q., Liu, Y., & Huang, W. (2018) · ACS Synthetic Biology · doi:10.1021/acssynbio.8b00202 · Cited by 36
SOX9 maintains human foetal lung tip progenitor state by enhancing WNT and RTK signalling
Sun, D., Llora Batlle, O., van den Ameele, J., Thomas, J. C., He, P., Lim, K., Tang, W., Xu, C., Meyer, K. B., Teichmann, S. A., et al. (2022) · The EMBO Journal · doi:10.15252/embj.2022111338 · Cited by 38
Holmgaard, A. B., Askou, A. L., Jensen, E. G., Alsing, S., Bak, R. O., Mikkelsen, J. G., & Corydon, T. J. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2020.09.032 · Cited by 36
Jones, L., Kumar, J., Mistry, A., Sankar Chittoor Mana, T., Perry, G., Reddy, V. P., & Obrenovich, M. (2019) · Biomedicines · doi:10.3390/biomedicines7020024 · Cited by 34
Technical considerations for the use of CRISPR/Cas9 in hematology research
Gundry, M. C., Dever, D. P., Yudovich, D., Bauer, D. E., Haas, S., Wilkinson, A. C., & Singbrant, S. (2017) · Experimental Hematology · doi:10.1016/j.exphem.2017.07.006 · Cited by 26
Gene Therapy for Inherited Retinal Degeneration
Arbabi, A., Liu, A., & Ameri, H. (2019) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2018.0087 · Cited by 30
Wang, X., Wei, Z., Gu, M., Zhu, L., Hai, C., Di, A., Wu, D., Bai, C., Su, G., Liu, X., et al. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232415707 · Cited by 29
Self-citationHoffman, H. K., Fernandez, M. V., Groves, N. S., Freed, E. O., & van Engelenburg, S. B. (2019) · Journal of Biological Chemistry · doi:10.1074/jbc.ra119.009372 · Cited by 28
Development of multiplexed orthogonal base editor (MOBE) systems
Cowan, Q. T., Gu, S., Gu, W., Ranzau, B. L., Simonson, T. S., & Komor, A. C. (2025) · Nature Biotechnology · doi:10.1038/s41587-024-02240-0 · Cited by 26
Development and application of prime editing in plants
Tingting, L., Jinpeng, Z., Xi, Y., Kejian, W., Yuchun, R., & Chun, W. (2023) · Rice Science · doi:10.1016/j.rsci.2023.07.005 · Cited by 21
Dissecting mammalian immunity through mutation
Siggs, O. M. (2014) · Immunology & Cell Biology · doi:10.1038/icb.2014.8 · Cited by 20
Rodent Genetic Models of Ah Receptor Signaling
Wilson, R. H., & Bradfield, C. A. (2021) · Drug Metabolism Reviews · doi:10.1080/03602532.2021.1955916 · Cited by 16
“AHR mutations induced by gene editing Within the past decade, the generation of model systems has been aided by the advancement of CRISPRbased gene editing techniques (Gasiunas et al. 2012; Jinek et al. 2012; Cong et al. 2013; Mali et al. 2013).”
MethodBoostMEC: predicting CRISPR-Cas9 cleavage efficiency through boosting models
Zarate, O. A., Yang, Y., Wang, X., & Wang, J.-P. (2022) · BMC Bioinformatics · doi:10.1186/s12859-022-04998-z · Cited by 17
Hybrid multitask learning reveals sequence features driving specificity in the CRISPR/Cas9 system
Vora, D. S., Yadav, S., & Sundar, D. (2023) · Biomolecules · doi:10.3390/biom13040641 · Cited by 17
Okada, K., Aoki, K., Tabei, T., Sugio, K., Imai, K., Bonkohara, Y., & Kamachi, Y. (2022) · Nucleic Acids Research · doi:10.1093/nar/gkac100 · Cited by 14
Grandchamp, N., Altémir, D., Philippe, S., Ursulet, S., Pilet, H., Serre, M.-C., Lenain, A., Serguera, C., Mallet, J., & Sarkis, C. (2014) · PLoS ONE · doi:10.1371/journal.pone.0099649 · Cited by 11
Chromatin structure and context-dependent sequence features control prime editing efficiency
Kim, S., Yuan, J. B., Woods, W. S., Newton, D. A., Perez-Pinera, P., & Song, J. S. (2023) · Frontiers in Genetics · doi:10.3389/fgene.2023.1222112 · Cited by 12
The history, landscape, and outlook of human cell line authentication and security
Harbut, E., Makris, Y., Pertsemlidis, A., & Bleris, L. (2024) · SLAS Discovery · doi:10.1016/j.slasd.2024.100194 · Cited by 8
Metadata
- Authors
- Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M.
- Affiliations
- Harvard University (US)
- Department of Genetics, Harvard Medical School, Boston, MA 02115, USA.
- Boston University (US)
- Corresponding author
- Mali, Prashant
- Yang, Luhan
- Church, George M.
- Venue
- Science
- Published
- 2013-02-15
- Volume
- 339
- Issue
- 6121
- Pages
- 823-826
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- PMC
- PMC3712628
- OpenAlex
- W2003171404
- Cited by
- 9,210
- References
- 46
- Citations by year
- 2026: 286 · 2025: 408 · 2024: 518 · 2023: 568 · 2022: 727 · 2021: 789 · 2020: 795 · 2019: 813 · 2018: 864 · 2017: 863 · 2016: 884 · 2015: 697 · 2014: 680 · 2013: 307
- FWCI
- 383.86
- Type
- journal article
- Language
- en
- Open access
- green
- europepmc.org
- Locations
- Science (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- RECERCAT (Consorci de Serveis Universitaris de Catalunya) (acceptedVersion)
- Repositori digital de la UPF (Universitat Pompeu Fabra) (submittedVersion)
- Funders
- National Human Genome Research Institute (P50HG005550)
- 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
- CRISPR/Cas9, guide RNA, human genome editing, AAVS1, multiplex genome editing, induced pluripotent stem cells, K562 cells, HEK293T cells, RNA-guided nucleases
- MeSH terms
- Chromosomes, Human, Pair 19, Codon, Exons, Genetic Engineering, Humans, RNA, Genome, Human, Gene Targeting, K562 Cells, DNA Cleavage, Inverted Repeat Sequences, Genetic Loci, Induced Pluripotent Stem Cells, Clustered Regularly Interspaced Short Palindromic Repeats
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