Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
Open accessAbstract
Clustered regularly interspaced short palindromic repeats (CRISPR) system provides adaptive immunity against plasmids and phages in prokaryotes. This system inspires the development of a powerful genome engineering tool, the CRISPR/CRISPR-associated nuclease 9 (CRISPR/Cas9) genome editing system. Due to its high efficiency and precision, the CRISPR/Cas9 technique has been employed to explore the functions of cancer-related genes, establish tumor-bearing animal models and probe drug targets, vastly increasing our understanding of cancer genomics. Here, we review current status of CRISPR/Cas9 gene editing technology in oncological research. We first explain the basic principles of CRISPR/Cas9 gene editing and introduce several new CRISPR-based gene editing modes. We next detail the rapid progress of CRISPR screening in revealing tumorigenesis, metastasis, and drug resistance mechanisms. In addition, we introduce CRISPR/Cas9 system delivery vectors and finally demonstrate the potential of CRISPR/Cas9 engineering to enhance the effect of adoptive T cell therapy (ACT) and reduce adverse reactions.
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For viral infections, CRISPR/Cas9 can be used to cut viral genomes and inhibit viral replication, such as in the case of HIV and Hepatitis B.
“For viral infections, CRISPR/Cas9 can be used to cut viral genomes and inhibit viral replication, such as in the case of HIV and Hepatitis B [7].”
From A Review of Research on CRISPR/Cas9 Gene Editing Technology in Disease Treatment (2024) · cites this paper as [7]
The Introduction (page unavailable) describes Cas9 cutting invading DNA as part of bacterial resistance to phage infection. That supports the general mechanism, but the passages found do not support its application to HIV or hepatitis B, or show inhibited replication of either virus; the Viral vectors section discusses delivery rather than treatment of those infections. (LLM, conf=0.82)
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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
Siegel, R. L., Miller, K. D., & Jemal, A. (2016) · CA: A Cancer Journal for Clinicians · doi:10.3322/caac.21332 · Cited by 16,003 · 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
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 · see citing works
Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia
Maude, S. L., Frey, N., Shaw, P. A., Aplenc, R., Barrett, D. M., Bunin, N. J., Chew, A., Gonzalez, V. E., Zheng, Z., Lacey, S. F., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407222 · Cited by 5,413 · cited 1× · see citing works
“CAR-T cell therapy against CD19 has achieved striking results in treatment of relapsed and refractory acute lymphoblastic leukemia, enabling 90% of patients to achieve complete remission [174].”
BackgroundGenome-scale CRISPR-Cas9 knockout screening in human cells
Shalem, O., Sanjana, N. E., Hartenian, E., Shi, X., Scott, D. A., Mikkelsen, T. S., Heckl, D., Ebert, B. L., Root, D. E., Doench, J. G., et al. (2014) · Science · doi:10.1126/science.1247005 · Cited by 5,234 · see citing works
Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
Qi, L. S., Larson, M. H., Gilbert, L. A., Doudna, J. A., Weissman, J. S., Arkin, A. P., & Lim, W. A. (2013) · Cell · doi:10.1016/j.cell.2013.02.022 · Cited by 5,016 · cited 1× · see citing works
“CRISPRi not only can effectively inhibit expression of multiple target genes in Escherichia coli simultaneously, but its effect also is reversible and does not appear to extend off-target [33].”
BackgroundProgrammable 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 · see citing works
A guide to cancer immunotherapy: from T cell basic science to clinical practice
Waldman, A. D., Fritz, J. M., & Lenardo, M. J. (2020) · Nature Reviews Immunology · doi:10.1038/s41577-020-0306-5 · Cited by 4,154 · see citing works
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
Gilbert, L. A., Larson, M. H., Morsut, L., Liu, Z., Brar, G. A., Torres, S. E., Stern-Ginossar, N., Brandman, O., Whitehead, E. H., Doudna, J. A., et al. (2013) · Cell · doi:10.1016/j.cell.2013.06.044 · Cited by 3,696 · see citing works
Design and development of polymers for gene delivery
Pack, D. W., Hoffman, A. S., Pun, S., & Stayton, P. S. (2005) · Nature Reviews Drug Discovery · doi:10.1038/nrd1775 · Cited by 2,568 · see citing works
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381 · see citing works
Crystal structure of Cas9 in complex with guide RNA and target DNA
Nishimasu, H., Ran, F. A., Hsu, P. D., Konermann, S., Shehata, S. I., Dohmae, N., Ishitani, R., Zhang, F., & Nureki, O. (2014) · Cell · doi:10.1016/j.cell.2014.02.001 · Cited by 2,306 · see citing works
Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection
Eyquem, J., Mansilla-Soto, J., Giavridis, T., van der Stegen, S. J. C., Hamieh, M., Cunanan, K. M., Odak, A., Gönen, M., & Sadelain, M. (2017) · Nature · doi:10.1038/nature21405 · Cited by 1,831 · cited 1× · see citing works
“Indeed, using the CRISPR system to direct CAR to the TRAC locus can avoid tonic CAR signal transduction and establish effective CAR internalization and reexpression after cells are exposed to antigen, effectively delaying differentiation and exhaustion of effector T cells [191].”
MethodBlank, C. U., Haining, W. N., Held, W., Hogan, P. G., Kallies, A., Lugli, E., Lynn, R. C., Philip, M., Rao, A., Restifo, N. P., et al. (2019) · Nature Reviews Immunology · doi:10.1038/s41577-019-0221-9 · Cited by 1,603 · see citing works
Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems
Nishida, K., Arazoe, T., Yachie, N., Banno, S., Kakimoto, M., Tabata, M., Mochizuki, M., Miyabe, A., Araki, M., Hara, K. Y., et al. (2016) · Science · doi:10.1126/science.aaf8729 · Cited by 1,349 · see citing works
Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
Walton, R. T., Christie, K. A., Whittaker, M. N., & Kleinstiver, B. P. (2020) · Science · doi:10.1126/science.aba8853 · Cited by 1,233 · see citing works
Delivering CRISPR: a review of the challenges and approaches
Lino, C. A., Harper, J. C., Carney, J. P., & Timlin, J. A. (2018) · Drug Delivery · doi:10.1080/10717544.2018.1474964 · Cited by 1,185 · see citing works
Pooled CRISPR screening with single-cell transcriptome readout.
Datlinger, P., Rendeiro, A. F., Schmidl, C., Krausgruber, T., Traxler, P., Klughammer, J., Schuster, L. C., Kuchler, A., Alpar, D., & Bock, C. (2017) · Nature Methods · doi:10.1038/nmeth.4177 · Cited by 1,096 · see citing works
c-Jun overexpression in CAR T cells induces exhaustion resistance
Lynn, R. C., Weber, E. W., Sotillo, E., Gennert, D., Xu, P., Good, Z., Anbunathan, H., Lattin, J., Jones, R., Tieu, V., et al. (2019) · Nature · doi:10.1038/s41586-019-1805-z · Cited by 891 · cited 1× · see citing works
“Similarly, CAR-T cells also acquire a depleted phenotype when entering the TME in vivo, which has become a major obstacle to CAR-T cell therapy [197, 198].”
BackgroundFinn, J. D., Smith, A. R., Patel, M. C., Shaw, L., Youniss, M. R., van Heteren, J., Dirstine, T., Ciullo, C., Lescarbeau, R., Seitzer, J., et al. (2018) · Cell Reports · doi:10.1016/j.celrep.2018.02.014 · Cited by 878 · see citing works
NR4A transcription factors limit CAR T cell function in solid tumours
Chen, J., López-Moyado, I. F., Seo, H., Lio, C.-W. J., Hempleman, L. J., Sekiya, T., Yoshimura, A., Scott-Browne, J. P., & Rao, A. (2019) · Nature · doi:10.1038/s41586-019-0985-x · Cited by 874 · cited 1× · see citing works
“Similarly, CAR-T cells also acquire a depleted phenotype when entering the TME in vivo, which has become a major obstacle to CAR-T cell therapy [197, 198].”
BackgroundIdentification of essential genes for cancer immunotherapy
Patel, S. J., Sanjana, N. E., Kishton, R. J., Eidizadeh, A., Vodnala, S. K., Cam, M., Gartner, J. J., Jia, L., Steinberg, S. M., Yamamoto, T. N., et al. (2017) · Nature · doi:10.1038/nature23477 · Cited by 870 · see citing works
In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9
Swiech, L., Heidenreich, M., Banerjee, A., Habib, N., Li, Y., Trombetta, J., Sur, M., & Zhang, F. (2015) · Nature Biotechnology · doi:10.1038/nbt.3055 · Cited by 772 · cited 1× · see citing works
“The AAV dual-vector system successfully targets a single gene or multiple genes in the mouse brain and characterizes the influences of genome modification on neurons [129], suggesting that that AAV-mediated genome editing can be applied to study brain gene function.”
BackgroundSelf-citationRupp, L. J., Schumann, K., Roybal, K. T., Gate, R. E., Ye, C. J., Lim, W. A., & Marson, A. (2017) · Scientific Reports · doi:10.1038/s41598-017-00462-8 · Cited by 757 · see citing works
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Rangarajan, S., Walsh, L., Lester, W., Perry, D., Madan, B., Laffan, M., Yu, H., Vettermann, C., Pierce, G. F., Wong, W. Y., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708483 · Cited by 694 · see citing works
CRISPR-mediated adaptive immune systems in bacteria and archaea
Sorek, R., Lawrence, C. M., & Wiedenheft, B. (2013) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-072911-172315 · Cited by 668 · see citing works
Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
Grünewald, J., Zhou, R., Garcia, S. P., Iyer, S., Lareau, C. A., Aryee, M. J., & Joung, J. K. (2019) · Nature · doi:10.1038/s41586-019-1161-z · Cited by 663 · see citing works
In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system
Maddalo, D., Manchado, E., Concepcion, C. P., Bonetti, C., Vidigal, J. A., Han, Y.-C., Ogrodowski, P., Crippa, A., Rekhtman, N., de Stanchina, E., et al. (2014) · Nature · doi:10.1038/nature13902 · Cited by 640 · see citing works
Genome-wide CRISPR Screens in Primary Human T Cells Reveal Key Regulators of Immune Function
Shifrut, E., Carnevale, J., Tobin, V., Roth, T. L., Woo, J. M., Bui, C. T., Li, P. J., Diolaiti, M. E., Ashworth, A., & Marson, A. (2018) · Cell · doi:10.1016/j.cell.2018.10.024 · Cited by 573 · see citing works
A public genome-scale lentiviral expression library of human ORFs
Yang, X., Boehm, J. S., Yang, X., Salehi-Ashtiani, K., Hao, T., Shen, Y., Lubonja, R., Thomas, S. R., Alkan, O., Bhimdi, T., et al. (2011) · Nature Methods · doi:10.1038/nmeth.1638 · Cited by 570 · see citing works
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3469 · Cited by 556 · cited 1× · see citing works
“When using AAVs for transfection, SpCas9 and sgRNA must be encoded on different vectors [124, 125].”
MethodEfficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9
Mandal, P. K., Ferreira, L. M. R., Collins, R., Meissner, T. B., Boutwell, C. L., Friesen, M., Vrbanac, V., Garrison, B. S., Stortchevoi, A., Bryder, D., et al. (2014) · Cell stem cell · doi:10.1016/j.stem.2014.10.004 · Cited by 475 · see citing works
Tisagenlecleucel — the first approved CAR-T-cell therapy: implications for payers and policy makers
Prasad, V. (2018) · Nature Reviews Clinical Oncology · doi:10.1038/nrclinonc.2017.156 · Cited by 229 · see citing works
Integration-deficient Lentiviral Vectors: A Slow Coming of Age
Wanisch, K., & Yáñez-Muñoz, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.122 · Cited by 222 · see citing works
Immune responses to adeno-associated virus and its recombinant vectors.
Sun, J. Y., Anand-Jawa, V, Chatterjee, S., & Wong, K. K. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302039 · Cited by 215 · see citing works
KEAP1 loss modulates sensitivity to kinase targeted therapy in lung cancer
Krall, E. B., Wang, B., Munoz, D. M., Ilic, N., Raghavan, S., Niederst, M. J., Yu, K., Ruddy, D. A., Aguirre, A. J., Kim, J. W., et al. (2017) · eLife · doi:10.7554/elife.18970 · Cited by 160 · see citing works
Flagged first, then by citations
Research works printed but not linked: 179
- [1]Labrie SJ Samson JE Moineau S Bacteriophage resistance mechanisms Nat Rev Microbiol 2010 8 317 327 20348932
- [2]Ishino Y Shinagawa H Makino K Amemura M Nakata A Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product J Bacteriol 1987 169 5429 5433 3316184 PMC213968
- [3]Pourcel C Salvignol G Vergnaud G CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies Microbiology (Reading) 2005 151 653 663 15758212
- [4]Bolotin A Quinquis B Sorokin A Ehrlich SD Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin Microbiology (Reading) 2005 151 2551 2561 16079334
- [5]Mojica FJ Díez-Villaseñor C García-Martínez J Soria E Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements J Mol Evol 2005 60 174 182 15791728
- [6]Barrangou R Fremaux C Deveau H Richards M Boyaval P Moineau S Romero DA Horvath P CRISPR provides acquired resistance against viruses in prokaryotes Science 2007 315 1709 1712 17379808
- [7]Tang TH Bachellerie JP Rozhdestvensky T Bortolin ML Huber H Drungowski M Elge T Brosius J Hüttenhofer A Identification of 86 candidates for small non-messenger RNAs from the archaeon Archaeoglobus fulgidus Proc Natl Acad Sci U S A 2002 99 7536 7541 12032318 PMC124276
- [8]Jansen R Embden JD Gaastra W Schouls LM Identification of genes that are associated with DNA repeats in prokaryotes Mol Microbiol 2002 43 1565 1575 11952905
- [10]Barrangou R Coûté-Monvoisin AC Stahl B Chavichvily I Damange F Romero DA Boyaval P Fremaux C Horvath P Genomic impact of CRISPR immunization against bacteriophages Biochem Soc Trans 2013 41 1383 1391 24256225
- [11]Fineran PC Charpentier E Memory of viral infections by CRISPR-Cas adaptive immune systems: acquisition of new information Virology 2012 434 202 209 23123013
- [12]Deltcheva E Chylinski K Sharma CM Gonzales K Chao Y Pirzada ZA Eckert MR Vogel J Charpentier E CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III Nature 2011 471 602 607 21455174 PMC3070239
- [14]Makarova KS Haft DH Barrangou R Brouns SJ Charpentier E Horvath P Moineau S Mojica FJ Wolf YI Yakunin AF Evolution and classification of the CRISPR-Cas systems Nat Rev Microbiol 2011 9 467 477 21552286 PMC3380444
- [15]Mali P Yang L Esvelt KM Aach J Guell M DiCarlo JE Norville JE Church GM RNA-guided human genome engineering via Cas9 Science 2013 339 823 826 23287722 PMC3712628
- [17]Bhaya D Davison M Barrangou R CRISPR-Cas systems in bacteria and archaea: versatile small RNAs for adaptive defense and regulation Annu Rev Genet 2011 45 273 297 22060043
- [18]Doudna JA Charpentier E Genome editing. The new frontier of genome engineering with CRISPR-Cas9 Science 2014 346 1258096 25430774
- [19]Sapranauskas R Gasiunas G Fremaux C Barrangou R Horvath P Siksnys V The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli Nucleic Acids Res 2011 39 9275 9282 21813460 PMC3241640
- [20]Shah SA Erdmann S Mojica FJ Garrett RA Protospacer recognition motifs: mixed identities and functional diversity RNA Biol 2013 10 891 899 23403393 PMC3737346
- [21]Sternberg SH Redding S Jinek M Greene EC Doudna JA DNA interrogation by the CRISPR RNA-guided endonuclease Cas9 Nature 2014 507 62 67 24476820 PMC4106473
- [23]Anders C Niewoehner O Duerst A Jinek M Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease Nature 2014 513 569 573 25079318 PMC4176945
- [24]Makarova KS Grishin NV Shabalina SA Wolf YI Koonin EV A putative RNA-interference-based immune system in prokaryotes: computational analysis of the predicted enzymatic machinery, functional analogies with eukaryotic RNAi, and hypothetical mechanisms of action Biol Direct 2006 1 7 16545108 PMC1462988
- [25]Gasiunas G Barrangou R Horvath P Siksnys V Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria Proc Natl Acad Sci U S A 2012 109 E2579 E2586 22949671 PMC3465414
- [26]Cyranoski D CRISPR gene-editing tested in a person for the first time Nature 2016 539 479 27882996
- [28]Anzalone AV Randolph PB Davis JR Sousa AA Koblan LW Levy JM Chen PJ Wilson C Newby GA Raguram A Liu DR Search-and-replace genome editing without double-strand breaks or donor DNA Nature 2019 576 149 157 31634902 PMC6907074
- [29]Liu Y Zou RS He S Nihongaki Y Li X Razavi S Wu B Ha T Very fast CRISPR on demand Science 2020 368 1265 1269 32527834 PMC7608738
- [30]Lieber MR Ma Y Pannicke U Schwarz K Mechanism and regulation of human non-homologous DNA end-joining Nat Rev Mol Cell Biol 2003 4 712 720 14506474
- [31]Cromie GA Connelly JC Leach DR Recombination at double-strand breaks and DNA ends: conserved mechanisms from phage to humans Mol Cell 2001 8 1163 1174 11779493
- [32]Rouet P Smih F Jasin M Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease Mol Cell Biol 1994 14 8096 8106 7969147 PMC359348
- [34]Perez-Pinera P Kocak DD Vockley CM Adler AF Kabadi AM Polstein LR Thakore PI Glass KA Ousterout DG Leong KW RNA-guided gene activation by CRISPR-Cas9-based transcription factors Nat Methods 2013 10 973 976 23892895 PMC3911785
- [36]Kosicki M Tomberg K Bradley A Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements Nat Biotechnol 2018 36 765 771 30010673 PMC6390938
- [37]Ihry RJ Worringer KA Salick MR Frias E Ho D Theriault K Kommineni S Chen J Sondey M Ye C p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells Nat Med 2018 24 939 946 29892062
- [38]Merkle FT Neuhausser WM Santos D Valen E Gagnon JA Maas K Sandoe J Schier AF Eggan K Efficient CRISPR-Cas9-mediated generation of knockin human pluripotent stem cells lacking undesired mutations at the targeted locus Cell Rep 2015 11 875 883 25937281 PMC5533178
- [39]Lin S Staahl BT Alla RK Doudna JA Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery Elife 2014 3 e04766 25497837 PMC4383097
- [40]Landrum MJ Lee JM Benson M Brown G Chao C Chitipiralla S Gu B Hart J Hoffman D Hoover J ClinVar: public archive of interpretations of clinically relevant variants Nucleic Acids Res 2016 44 D862 D868 26582918 PMC4702865
- [41]Komor AC Badran AH Liu DR CRISPR-based technologies for the manipulation of eukaryotic genomes Cell 2017 168 20 36 27866654 PMC5235943
- [43]Rees HA Liu DR Base editing: precision chemistry on the genome and transcriptome of living cells Nat Rev Genet 2018 19 770 788 30323312 PMC6535181
- [45]Harris RS Petersen-Mahrt SK Neuberger MS RNA editing enzyme APOBEC1 and some of its homologs can act as DNA mutators Mol Cell 2002 10 1247 1253 12453430
- [46]Jiang F Doudna JA CRISPR-Cas9 structures and mechanisms Annu Rev Biophys 2017 46 505 529 28375731
- [47]Li Z Pearlman AH Hsieh P DNA mismatch repair and the DNA damage response DNA Repair (Amst) 2016 38 94 101 26704428 PMC4740233
- [48]Kunz C Saito Y Schär P DNA repair in mammalian cells: mismatched repair: variations on a theme Cell Mol Life Sci 2009 66 1021 1038 19153655 PMC11131451
- [49]Mol CD Arvai AS Sanderson RJ Slupphaug G Kavli B Krokan HE Mosbaugh DW Tainer JA Crystal structure of human uracil-DNA glycosylase in complex with a protein inhibitor: protein mimicry of DNA Cell 1995 82 701 708 7671300
- [50]Komor AC Zhao KT Packer MS Gaudelli NM Waterbury AL Koblan LW Kim YB Badran AH Liu DR Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity Sci Adv 2017 3 eaao4774 28875174 PMC5576876
- [51]Zafra MP Schatoff EM Katti A Foronda M Breinig M Schweitzer AY Simon A Han T Goswami S Montgomery E Optimized base editors enable efficient editing in cells, organoids and mice Nat Biotechnol 2018 36 888 893 29969439 PMC6130889
- [52]Lewis CA Jr Crayle J Zhou S Swanstrom R Wolfenden R Cytosine deamination and the precipitous decline of spontaneous mutation during Earth’s history Proc Natl Acad Sci U S A 2016 113 8194 8199 27382162 PMC4961170
- [53]Yasui M Suenaga E Koyama N Masutani C Hanaoka F Gruz P Shibutani S Nohmi T Hayashi M Honma M Miscoding properties of 2′-deoxyinosine, a nitric oxide-derived DNA Adduct, during translesion synthesis catalyzed by human DNA polymerases J Mol Biol 2008 377 1015 1023 18304575
- [54]Zheng Y Lorenzo C Beal PA DNA editing in DNA/RNA hybrids by adenosine deaminases that act on RNA Nucleic Acids Res 2017 45 3369 3377 28132026 PMC5389660
- [55]Newby GA Yen JS Woodard KJ Mayuranathan T Lazzarotto CR Li Y Sheppard-Tillman H Porter SN Yao Y Mayberry K Base editing of haematopoietic stem cells rescues sickle cell disease in mice Nature 2021 595 295 302 34079130 PMC8266759
- [56]Wang Q Yang J Zhong Z Vanegas JA Gao X Kolomeisky AB A general theoretical framework to design base editors with reduced bystander effects Nat Commun 2021 12 6529 34764246 PMC8586357
- [57]Kim HS Jeong YK Hur JK Kim JS Bae S Adenine base editors catalyze cytosine conversions in human cells Nat Biotechnol 2019 37 1145 1148 31548727
- [58]Newby GA Liu DR In vivo somatic cell base editing and prime editing Mol Ther 2021 29 3107 3124 34509669 PMC8571176
- [59]Kim YB Komor AC Levy JM Packer MS Zhao KT Liu DR Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions Nat Biotechnol 2017 35 371 376 28191901 PMC5388574
- [60]Hu JH Miller SM Geurts MH Tang W Chen L Sun N Zeina CM Gao X Rees HA Lin Z Liu DR Evolved Cas9 variants with broad PAM compatibility and high DNA specificity Nature 2018 556 57 63 29512652 PMC5951633
- [62]Zuo E Sun Y Wei W Yuan T Ying W Sun H Yuan L Steinmetz LM Li Y Yang H Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos Science 2019 364 289 292 30819928 PMC7301308
- [64]Jin S Lin Q Luo Y Zhu Z Liu G Li Y Chen K Qiu JL Gao C Genome-wide specificity of prime editors in plants Nat Biotechnol 2021 39 1292 1299 33859403
- [65]Kim DY Moon SB Ko JH Kim YS Kim D Unbiased investigation of specificities of prime editing systems in human cells Nucleic Acids Res 2020 48 10576 10589 32941652 PMC7544197
- [66]Yin H Xue W Anderson DG CRISPR-Cas: a tool for cancer research and therapeutics Nat Rev Clin Oncol 2019 16 281 295 30664678
- [69]Wang T Wei JJ Sabatini DM Lander ES Genetic screens in human cells using the CRISPR-Cas9 system Science 2014 343 80 84 24336569 PMC3972032
- [70]Zhou Y Zhu S Cai C Yuan P Li C Huang Y Wei W High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells Nature 2014 509 487 491 24717434
- [71]Yamauchi T Masuda T Canver MC Seiler M Semba Y Shboul M Al-Raqad M Maeda M Schoonenberg VAC Cole MA Genome-wide CRISPR-Cas9 screen identifies leukemia-specific dependence on a pre-mRNA metabolic pathway regulated by DCPS Cancer Cell 2018 33 386 400.e385 29478914 PMC5849534
- [72]Tzelepis K Koike-Yusa H De Braekeleer E Li Y Metzakopian E Dovey OM Mupo A Grinkevich V Li M Mazan M A CRISPR dropout screen identifies genetic vulnerabilities and therapeutic targets in acute myeloid leukemia Cell Rep 2016 17 1193 1205 27760321 PMC5081405
- [75]Benci JL Johnson LR Choa R Xu Y Qiu J Zhou Z Xu B Ye D Nathanson KL June CH Opposing functions of interferon coordinate adaptive and innate immune responses to cancer immune checkpoint blockade Cell 2019 178 933 948.e914 31398344 PMC6830508
- [76]Cursons J Souza-Fonseca-Guimaraes F Foroutan M Anderson A Hollande F Hediyeh-Zadeh S Behren A Huntington ND Davis MJ A gene signature predicting natural killer cell infiltration and improved survival in melanoma patients Cancer Immunol Res 2019 7 1162 1174 31088844
- [77]Freeman AJ Vervoort SJ Ramsbottom KM Kelly MJ Michie J Pijpers L Johnstone RW Kearney CJ Oliaro J Natural killer cells suppress T cell-associated tumor immune evasion Cell Rep 2019 28 2784 2794.e2785 31509742
- [78]Sheffer M, Lowry E, Beelen N, Borah M, Amara SNA, Mader CC, et al. Genome-scale screens identify factors regulating tumor cell responses to natural killer cells. Nat Genet. 2021;53:1196–206. 34253920
- [80]Nieto MA Huang RY Jackson RA Thiery JP EMT: 2016 Cell 2016 166 21 45 27368099
- [81]McFaline-Figueroa JL Hill AJ Qiu X Jackson D Shendure J Trapnell C A pooled single-cell genetic screen identifies regulatory checkpoints in the continuum of the epithelial-to-mesenchymal transition Nat Genet 2019 51 1389 1398 31477929 PMC6756480
- [82]Shu S Lin CY He HH Witwicki RM Tabassum DP Roberts JM Janiszewska M Huh SJ Liang Y Ryan J Response and resistance to BET bromodomain inhibitors in triple-negative breast cancer Nature 2016 529 413 417 26735014 PMC4854653
- [83]Shu S Wu HJ Ge JY Zeid R Harris IS Jovanović B Murphy K Wang B Qiu X Endress JE Synthetic lethal and resistance interactions with BET bromodomain inhibitors in triple-negative breast cancer Mol Cell 2020 78 1096 1113.e1098 32416067 PMC7306005
- [85]Schumann K Lin S Boyer E Simeonov DR Subramaniam M Gate RE Haliburton GE Ye CJ Bluestone JA Doudna JA Marson A Generation of knock-in primary human T cells using Cas9 ribonucleoproteins Proc Natl Acad Sci U S A 2015 112 10437 10442 26216948 PMC4547290
- [86]Shang W Jiang Y Boettcher M Ding K Mollenauer M Liu Z Wen X Liu C Hao P Zhao S Genome-wide CRISPR screen identifies FAM49B as a key regulator of actin dynamics and T cell activation Proc Natl Acad Sci U S A 2018 115 E4051 e4060 29632189 PMC5924929
- [88]Schumann K Raju SS Lauber M Kolb S Shifrut E Cortez JT Skartsis N Nguyen VQ Woo JM Roth TL Functional CRISPR dissection of gene networks controlling human regulatory T cell identity Nat Immunol 2020 21 1456 1466 32989329 PMC7577958
- [89]Li F Ng WL Luster TA Hu H Sviderskiy VO Dowling CM Hollinshead KER Zouitine P Zhang H Huang Q Epigenetic CRISPR screens identify Npm1 as a therapeutic vulnerability in non-small cell lung cancer Cancer Res 2020 80 3556 3567 32646968 PMC7493834
- [90]Shi S Gu S Han T Zhang W Huang L Li Z Pan D Fu J Ge J Brown M Inhibition of MAN2A1 enhances the immune response to anti-PD-L1 in human tumors Clin Cancer Res 2020 26 5990 6002 32723834 PMC8500537
- [91]Lian B Pei YC Jiang YZ Xue MZ Li DQ Li XG Zheng YZ Liu XY Qiao F Sun WL Truncated HDAC9 identified by integrated genome-wide screen as the key modulator for paclitaxel resistance in triple-negative breast cancer Theranostics 2020 10 11092 11109 33042272 PMC7532680
- [92]Miller FR Medina D Heppner GH Preferential growth of mammary tumors in intact mammary fatpads Cancer Res 1981 41 3863 3867 7284994
- [93]Erstad DJ Sojoodi M Taylor MS Ghoshal S Razavi AA Graham-O'Regan KA Bardeesy N Ferrone CR Lanuti M Caravan P Orthotopic and heterotopic murine models of pancreatic cancer and their different responses to FOLFIRINOX chemotherapy Dis Model Mech 2018 11 7 dmm034793 29903803 PMC6078400
- [94]Qin X Li J Wang S Lv J Luan F Liu Y Chen Y Chen X Zhao Y Zhu J Serotonin/HTR1E signaling blocks chronic stress-promoted progression of ovarian cancer Theranostics 2021 11 6950 6965 34093864 PMC8171092
- [95]Mallya K Gautam SK Aithal A Batra SK Jain M Modeling pancreatic cancer in mice for experimental therapeutics Biochim Biophys Acta Rev Cancer 2021 1876 188554 33945847 PMC8570383
- [96]Ishizuka JJ Manguso RT Cheruiyot CK Bi K Panda A Iracheta-Vellve A Miller BC Du PP Yates KB Dubrot J Loss of ADAR1 in tumours overcomes resistance to immune checkpoint blockade Nature 2019 565 43 48 30559380 PMC7241251
- [97]Hausser HJ Brenner RE Phenotypic instability of Saos-2 cells in long-term culture Biochem Biophys Res Commun 2005 333 216 222 15939397
- [98]Hidalgo M Amant F Biankin AV Budinská E Byrne AT Caldas C Clarke RB de Jong S Jonkers J Mælandsmo GM Patient-derived xenograft models: an emerging platform for translational cancer research Cancer Discov 2014 4 998 1013 25185190 PMC4167608
- [99]Tentler JJ Tan AC Weekes CD Jimeno A Leong S Pitts TM Arcaroli JJ Messersmith WA Eckhardt SG Patient-derived tumour xenografts as models for oncology drug development Nat Rev Clin Oncol 2012 9 338 350 22508028 PMC3928688
- [101]Wei X Yang J Adair SJ Ozturk H Kuscu C Lee KY Kane WJ O'Hara PE Liu D Demirlenk YM Targeted CRISPR screening identifies PRMT5 as synthetic lethality combinatorial target with gemcitabine in pancreatic cancer cells Proc Natl Acad Sci U S A 2020 117 28068 28079 33097661 PMC7668168
- [102]Lin S, Larrue C, Scheidegger NK, Seong BKA, Dharia NV, Kuljanin M, et al. An in vivo CRISPR screening platform for prioritizing therapeutic targets in AML. Cancer Discov. 2021. 10.1158/2159-8290.CD-20-1851. PMC8831447 34531254 · DOI
- [103]Kudo M Finn RS Qin S Han KH Ikeda K Piscaglia F Baron A Park JW Han G Jassem J Lenvatinib versus sorafenib in first-line treatment of patients with unresectable hepatocellular carcinoma: a randomised phase 3 non-inferiority trial Lancet 2018 391 1163 1173 29433850
- [104]Jin H Shi Y Lv Y Yuan S Ramirez CFA Lieftink C Wang L Wang S Wang C Dias MH EGFR activation limits the response of liver cancer to lenvatinib Nature 2021 595 730 734 34290403
- [105]Wilbie D Walther J Mastrobattista E Delivery aspects of CRISPR/Cas for in vivo genome editing Acc Chem Res 2019 52 1555 1564 31099553 PMC6584901
- [106]Kay MA Glorioso JC Naldini L Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics Nat Med 2001 7 33 40 11135613
- [107]Bessis N GarciaCozar FJ Boissier MC Immune responses to gene therapy vectors: influence on vector function and effector mechanisms Gene Ther 2004 11 Suppl 1 S10 S17 15454952
- [108]Samulski RJ Zhu X Xiao X Brook JD Housman DE Epstein N Hunter LA Targeted integration of adeno-associated virus (AAV) into human chromosome 19 EMBO J 1991 10 3941 3950 1657596 PMC453134
- [109]Yan J Kang DD Turnbull G Dong Y Delivery of CRISPR-Cas9 system for screening and editing RNA binding proteins in cancer Adv Drug Deliv Rev 2021 180 114042 34767864 PMC8724402
- [110]Maggio I Zittersteijn HA Wang Q Liu J Janssen JM Ojeda IT van der Maarel SM Lankester AC Hoeben RC Gonçalves M Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components Gene Ther 2020 27 209 225 31900423 PMC7253353
- [111]Palmer D Ng P Improved system for helper-dependent adenoviral vector production Mol Ther 2003 8 846 852 14599819
- [112]Jager L Ehrhardt A Persistence of high-capacity adenoviral vectors as replication-defective monomeric genomes in vitro and in murine liver Hum Gene Ther 2009 20 883 896 19364285
- [113]Byrnes AP Rusby JE Wood MJ Charlton HM Adenovirus gene transfer causes inflammation in the brain Neuroscience 1995 66 1015 1024 7651606
- [114]Yang Y Greenough K Wilson JM Transient immune blockade prevents formation of neutralizing antibody to recombinant adenovirus and allows repeated gene transfer to mouse liver Gene Ther 1996 3 412 420 9156802
- [115]Beer SJ Matthews CB Stein CS Ross BD Hilfinger JM Davidson BL Poly (lactic-glycolic) acid copolymer encapsulation of recombinant adenovirus reduces immunogenicity in vivo Gene Ther 1998 5 740 746 9747453
- [116]Tuveson DA Jacks T Technologically advanced cancer modeling in mice Curr Opin Genet Dev 2002 12 105 110 11790563
- [118]Wang D Mou H Li S Li Y Hough S Tran K Li J Yin H Anderson DG Sontheimer EJ Adenovirus-mediated somatic genome editing of Pten by CRISPR/Cas9 in mouse liver in spite of Cas9-specific immune responses Hum Gene Ther 2015 26 432 442 26086867 PMC4509492
- [119]Huang J Chen M Whitley MJ Kuo HC Xu ES Walens A Mowery YM Van Mater D Eward WC Cardona DM Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma Nat Commun 2017 8 15999 28691711 PMC5508130
- [120]Wang D Tai PWL Gao G Adeno-associated virus vector as a platform for gene therapy delivery Nat Rev Drug Discov 2019 18 358 378 30710128 PMC6927556
- [121]Luo J Luo Y Sun J Zhou Y Zhang Y Yang X Adeno-associated virus-mediated cancer gene therapy: current status Cancer Lett 2015 356 347 356 25444906 PMC4259838
- [122]Nelson CE Gersbach CA Engineering delivery vehicles for genome editing Annu Rev Chem Biomol Eng 2016 7 637 662 27146557
- [123]Dong JY Fan PD Frizzell RA Quantitative analysis of the packaging capacity of recombinant adeno-associated virus Hum Gene Ther 1996 7 2101 2112 8934224
- [124]Senís E Fatouros C Große S Wiedtke E Niopek D Mueller AK Börner K Grimm D CRISPR/Cas9-mediated genome engineering: an adeno-associated viral (AAV) vector toolbox Biotechnol J 2014 9 1402 1412 25186301
- [127]Huttner NA Girod A Perabo L Edbauer D Kleinschmidt JA Büning H Hallek M Genetic modifications of the adeno-associated virus type 2 capsid reduce the affinity and the neutralizing effects of human serum antibodies Gene Ther 2003 10 2139 2147 14625569
- [128]Vakulskas CA Behlke MA Evaluation and reduction of CRISPR off-target cleavage events Nucleic Acid Ther 2019 29 167 174 31107154 PMC6686686
- [130]Winters IP Chiou SH Paulk NK McFarland CD Lalgudi PV Ma RK Lisowski L Connolly AJ Petrov DA Kay MA Winslow MM Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity Nat Commun 2017 8 2053 29233960 PMC5727199
- [131]Chow RD Guzman CD Wang G Schmidt F Youngblood MW Ye L Errami Y Dong MB Martinez MA Zhang S AAV-mediated direct in vivo CRISPR screen identifies functional suppressors in glioblastoma Nat Neurosci 2017 20 1329 1341 28805815 PMC5614841
- [132]Naldini L Lentiviruses as gene transfer agents for delivery to non-dividing cells Curr Opin Biotechnol 1998 9 457 463 9821272
- [133]Kantor B Bailey RM Wimberly K Kalburgi SN Gray SJ Methods for gene transfer to the central nervous system Adv Genet 2014 87 125 197 25311922 PMC4519829
- [134]Ling S Yang S Hu X Yin D Dai Y Qian X Wang D Pan X Hong J Sun X Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice Nat Biomed Eng 2021 5 144 156 33398131
- [136]Kim W Lee S Kim HS Song M Cha YH Kim YH Shin J Lee ES Joo Y Song JJ Targeting mutant KRAS with CRISPR-Cas9 controls tumor growth Genome Res 2018 28 374 382 PMC5848616 29326299
- [137]Chen SH Hsieh YY Tzeng HE Lin CY Hsu KW Chiang YS Lin SM Su MJ Hsieh WS Lee CH ABL genomic editing sufficiently abolishes oncogenesis of human chronic myeloid leukemia cells in vitro and in vivo Cancers (Basel) 2020 12 6 1399 PMC7352505 32485885
- [138]High KA Turning genes into medicines-what have we learned from gene therapy drug development in the past decade? Nat Commun 2020 11 5821 33199678 PMC7669843
- [139]Baum C Kustikova O Modlich U Li Z Fehse B Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors Hum Gene Ther 2006 17 253 263 16544975
- [140]Waehler R Russell SJ Curiel DT Engineering targeted viral vectors for gene therapy Nat Rev Genet 2007 8 573 587 17607305 PMC7097627
- [142]Pahle J Walther W Vectors and strategies for nonviral cancer gene therapy Expert Opin Biol Ther 2016 16 443 461 26761200
- [143]Witzigmann D Kulkarni JA Leung J Chen S Cullis PR van der Meel R Lipid nanoparticle technology for therapeutic gene regulation in the liver Adv Drug Deliv Rev 2020 159 344 363 32622021 PMC7329694
- [144]Sercombe L Veerati T Moheimani F Wu SY Sood AK Hua S Advances and challenges of liposome assisted drug delivery Front Pharmacol 2015 6 286 26648870 PMC4664963
- [145]Mehnert W Mäder K Solid lipid nanoparticles: production, characterization and applications Adv Drug Deliv Rev 2001 47 165 196 11311991
- [146]Pardi N Hogan MJ Pelc RS Muramatsu H Andersen H DeMaso CR Dowd KA Sutherland LL Scearce RM Parks R Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination Nature 2017 543 248 251 28151488 PMC5344708
- [148]Cheng Q Wei T Farbiak L Johnson LT Dilliard SA Siegwart DJ Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing Nat Nanotechnol 2020 15 313 320 32251383 PMC7735425
- [149]Song R Murphy M Li C Ting K Soo C Zheng Z Current development of biodegradable polymeric materials for biomedical applications Drug Des Devel Ther 2018 12 3117 3145 30288019 PMC6161720
- [150]Yan M Du J Gu Z Liang M Hu Y Zhang W Priceman S Wu L Zhou ZH Liu Z A novel intracellular protein delivery platform based on single-protein nanocapsules Nat Nanotechnol 2010 5 48 53 19935648
- [151]Chen G Abdeen AA Wang Y Shahi PK Robertson S Xie R Suzuki M Pattnaik BR Saha K Gong S A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing Nat Nanotechnol 2019 14 974 980 31501532 PMC6778035
- [152]Wan T Chen Y Pan Q Xu X Kang Y Gao X Huang F Wu C Ping Y Genome editing of mutant KRAS through supramolecular polymer-mediated delivery of Cas9 ribonucleoprotein for colorectal cancer therapy J Control Release 2020 322 236 247 32169537
- [153]Guo P Yang J Huang J Auguste DT Moses MA Therapeutic genome editing of triple-negative breast tumors using a noncationic and deformable nanolipogel Proc Natl Acad Sci U S A 2019 116 18295 18303 31451668 PMC6744870
- [154]Deng H Tan S Gao X Zou C Xu C Tu K Song Q Fan F Huang W Zhang Z Cdk5 knocking out mediated by CRISPR-Cas9 genome editing for PD-L1 attenuation and enhanced antitumor immunity Acta Pharm Sin B 2020 10 358 373 32082979 PMC7016277
- [155]Gindy ME Prud'homme RK Multifunctional nanoparticles for imaging, delivery and targeting in cancer therapy Expert Opin Drug Deliv 2009 6 865 878 19637974
- [156]Bowman MC Ballard TE Ackerson CJ Feldheim DL Margolis DM Melander C Inhibition of HIV fusion with multivalent gold nanoparticles J Am Chem Soc 2008 130 6896 6897 18473457 PMC2916654
- [157]Chompoosor A Saha K Ghosh PS Macarthy DJ Miranda OR Zhu ZJ Arcaro KF Rotello VM The role of surface functionality on acute cytotoxicity, ROS generation and DNA damage by cationic gold nanoparticles Small 2010 6 2246 2249 20818619 PMC3016093
- [158]De Jong WH Hagens WI Krystek P Burger MC Sips AJ Geertsma RE Particle size-dependent organ distribution of gold nanoparticles after intravenous administration Biomaterials 2008 29 1912 1919 18242692
- [159]Takeuchi I Onaka H Makino K Biodistribution of colloidal gold nanoparticles after intravenous injection: effects of PEGylation at the same particle size Biomed Mater Eng 2018 29 205 215 29457594
- [160]Ahmad S Zamry AA Tan HT Wong KK Lim J Mohamud R Targeting dendritic cells through gold nanoparticles: a review on the cellular uptake and subsequent immunological properties Mol Immunol 2017 91 123 133 28898717
- [162]Lee K Conboy M Park HM Jiang F Kim HJ Dewitt MA Mackley VA Chang K Rao A Skinner C Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair Nat Biomed Eng 2017 1 889 901 29805845 PMC5968829
- [163]Yen HJ Hsu SH Tsai CL Cytotoxicity and immunological response of gold and silver nanoparticles of different sizes Small 2009 5 1553 1561 19326357
- [164]Bastús NG Sánchez-Tilló E Pujals S Farrera C López C Giralt E Celada A Lloberas J Puntes V Homogeneous conjugation of peptides onto gold nanoparticles enhances macrophage response ACS Nano 2009 3 1335 1344 19489561
- [165]Lee JY Park W Yi DK Immunostimulatory effects of gold nanorod and silica-coated gold nanorod on RAW 264.7 mouse macrophages Toxicol Lett 2012 209 51 57 22155353
- [166]Zhang B CRISPR/Cas gene therapy J Cell Physiol 2021 236 2459 2481 32959897
- [167]Hirsch T Rothoeft T Teig N Bauer JW Pellegrini G De Rosa L Scaglione D Reichelt J Klausegger A Kneisz D Regeneration of the entire human epidermis using transgenic stem cells Nature 2017 551 327 332 29144448 PMC6283270
- [170]Russell S Bennett J Wellman JA Chung DC Yu ZF Tillman A Wittes J Pappas J Elci O McCague S Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial Lancet 2017 390 849 860 28712537 PMC5726391
- [171]Tebas P Stein D Tang WW Frank I Wang SQ Lee G Spratt SK Surosky RT Giedlin MA Nichol G Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV N Engl J Med 2014 370 901 910 24597865 PMC4084652
- [173]Yong CSM Dardalhon V Devaud C Taylor N Darcy PK Kershaw MH CAR T-cell therapy of solid tumors Immunol Cell Biol 2017 95 356 363 28003642
- [175]Kershaw MH Westwood JA Parker LL Wang G Eshhar Z Mavroukakis SA White DE Wunderlich JR Canevari S Rogers-Freezer L A phase I study on adoptive immunotherapy using gene-modified T cells for ovarian cancer Clin Cancer Res 2006 12 6106 6115 17062687 PMC2154351
- [176]Lamers CH Sleijfer S Vulto AG Kruit WH Kliffen M Debets R Gratama JW Stoter G Oosterwijk E Treatment of metastatic renal cell carcinoma with autologous T-lymphocytes genetically retargeted against carbonic anhydrase IX: first clinical experience J Clin Oncol 2006 24 e20 e22 16648493
- [177]Fix SM Jazaeri AA Hwu P Applications of CRISPR genome editing to advance the next generation of adoptive cell therapies for cancer Cancer Discov 2021 11 560 574 33563662 PMC8193798
- [178]Maus MV Fraietta JA Levine BL Kalos M Zhao Y June CH Adoptive immunotherapy for cancer or viruses Annu Rev Immunol 2014 32 189 225 24423116 PMC4533835
- [179]Sadelain M Rivière I Brentjens R Targeting tumours with genetically enhanced T lymphocytes Nat Rev Cancer 2003 3 35 45 12509765
- [180]Neelapu SS Tummala S Kebriaei P Wierda W Gutierrez C Locke FL Komanduri KV Lin Y Jain N Daver N Chimeric antigen receptor T-cell therapy - assessment and management of toxicities Nat Rev Clin Oncol 2018 15 47 62 28925994 PMC6733403
- [181]Brocker T Karjalainen K Signals through T cell receptor-zeta chain alone are insufficient to prime resting T lymphocytes J Exp Med 1995 181 1653 1659 7722445 PMC2192006
- [182]Krause A Guo HF Latouche JB Tan C Cheung NK Sadelain M Antigen-dependent CD28 signaling selectively enhances survival and proliferation in genetically modified activated human primary T lymphocytes J Exp Med 1998 188 619 626 9705944 PMC2213361
- [183]Sadelain M Brentjens R Rivière I The basic principles of chimeric antigen receptor design Cancer Discov 2013 3 388 398 23550147 PMC3667586
- [184]Wang X Rivière I Clinical manufacturing of CAR T cells: foundation of a promising therapy Mol Ther Oncolytics 2016 3 16015 27347557 PMC4909095
- [186]Köhl U Arsenieva S Holzinger A Abken H CAR T cells in trials: recent achievements and challenges that remain in the production of modified T cells for clinical applications Hum Gene Ther 2018 29 559 568 29620951
- [187]Gill S June CH Going viral: chimeric antigen receptor T-cell therapy for hematological malignancies Immunol Rev 2015 263 68 89 25510272
- [188]Alcover A Alarcón B Di Bartolo V Cell biology of T cell receptor expression and regulation Annu Rev Immunol 2018 36 103 125 29261409
- [189]Torikai H Reik A Liu PQ Zhou Y Zhang L Maiti S Huls H Miller JC Kebriaei P Rabinovich B A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR Blood 2012 119 5697 5705 22535661 PMC3382929
- [190]Qasim W Zhan H Samarasinghe S Adams S Amrolia P Stafford S Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells Sci Transl Med. 2017 9 374 eaaj2013 28123068
- [192]Jackson HJ Rafiq S Brentjens RJ Driving CAR T-cells forward Nat Rev Clin Oncol 2016 13 370 383 27000958 PMC5529102
- [193]Wei SC Duffy CR Allison JP Fundamental mechanisms of immune checkpoint blockade therapy Cancer Discov 2018 8 1069 1086 30115704
- [195]Choi BD Yu X Castano AP Darr H Henderson DB Bouffard AA Larson RC Scarfò I Bailey SR Gerhard GM CRISPR-Cas9 disruption of PD-1 enhances activity of universal EGFRvIII CAR T cells in a preclinical model of human glioblastoma J Immunother Cancer 2019 7 304 31727131 PMC6857271
- [199]Good CR Aznar MA Kuramitsu S Samareh P Agarwal S Donahue G Ishiyama K Wellhausen N Rennels AK Ma Y An NK-like CAR T cell transition in CAR T cell dysfunction Cell 2021 184 25 6081 6100.e26 34861191 PMC8827167
- [200]Bonifant CL Jackson HJ Brentjens RJ Curran KJ Toxicity and management in CAR T-cell therapy Mol Ther Oncolytics 2016 3 16011 27626062 PMC5008265
- [201]Morris EC Stauss HJ Optimizing T-cell receptor gene therapy for hematologic malignancies Blood 2016 127 3305 3311 27207802 PMC5316910
- [202]Dembić Z Haas W Weiss S McCubrey J Kiefer H von Boehmer H Steinmetz M Transfer of specificity by murine alpha and beta T-cell receptor genes Nature 1986 320 232 238 2421164
- [203]Amir AL van der Steen DM van Loenen MM Hagedoorn RS de Boer R Kester MD de Ru AH Lugthart GJ van Kooten C Hiemstra PS PRAME-specific Allo-HLA-restricted T cells with potent antitumor reactivity useful for therapeutic T-cell receptor gene transfer Clin Cancer Res 2011 17 5615 5625 21771875
- [204]van Loenen MM de Boer R Amir AL Hagedoorn RS Volbeda GL Willemze R van Rood JJ Falkenburg JH Heemskerk MH Mixed T cell receptor dimers harbor potentially harmful neoreactivity Proc Natl Acad Sci U S A 2010 107 10972 10977 20534461 PMC2890759
- [205]Bendle GM Linnemann C Hooijkaas AI Bies L de Witte MA Jorritsma A Kaiser AD Pouw N Debets R Kieback E Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy Nat Med 2010 16 565 570 20400962
- [206]Ahmadi M King JW Xue SA Voisine C Holler A Wright GP Waxman J Morris E Stauss HJ CD3 limits the efficacy of TCR gene therapy in vivo Blood 2011 118 3528 3537 21750319
- [207]Provasi E Genovese P Lombardo A Magnani Z Liu PQ Reik A Chu V Paschon DE Zhang L Kuball J Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer Nat Med 2012 18 807 815 22466705 PMC5019824
- [208]Legut M Dolton G Mian AA Ottmann OG Sewell AK CRISPR-mediated TCR replacement generates superior anticancer transgenic T cells Blood 2018 131 311 322 29122757 PMC5774207
- [209]Morton LT Reijmers RM Wouters AK Kweekel C Remst DFG Pothast CR Falkenburg JHF Heemskerk MHM Simultaneous deletion of endogenous TCRαβ for TCR gene therapy creates an improved and safe cellular therapeutic Mol Ther 2020 28 64 74 31636040 PMC6953894
- [210]Stadtmauer EA Fraietta JA Davis MM Cohen AD Weber KL Lancaster E Mangan PA Kulikovskaya I Gupta M Chen F CRISPR-engineered T cells in patients with refractory cancer Science 2020 367 6481 eaba7365 32029687 PMC11249135
- [211]Zhang C Peng Y Hublitz P Zhang H Dong T Genetic abrogation of immune checkpoints in antigen-specific cytotoxic T-lymphocyte as a potential alternative to blockade immunotherapy Sci Rep 2018 8 5549 29615718 PMC5882910
- [212]Zhang W Shi L Zhao Z Du P Ye X Li D Cai Z Han J Cai J Disruption of CTLA-4 expression on peripheral blood CD8 + T cell enhances anti-tumor efficacy in bladder cancer Cancer Chemother Pharmacol 2019 83 911 920 30848330
- [213]Johnson LA Morgan RA Dudley ME Cassard L Yang JC Hughes MS Kammula US Royal RE Sherry RM Wunderlich JR Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen Blood 2009 114 535 546 19451549 PMC2929689
- [214]Bulcha JT Wang Y Ma H Tai PWL Gao G Viral vector platforms within the gene therapy landscape Signal Transduct Target Ther 2021 6 53 33558455 PMC7868676
- [215]Xu X Liu C Wang Y Koivisto O Zhou J Shu Y Zhang H Nanotechnology-based delivery of CRISPR/Cas9 for cancer treatment Adv Drug Deliv Rev 2021 176 113891 34324887
- [216]Ehrke-Schulz E Heinemann S Schulte L Schiwon M Ehrhardt A Adenoviral vectors armed with PAPILLOMAVIRUs oncogene specific CRISPR/Cas9 kill human-papillomavirus-induced cervical cancer cells Cancers (Basel) 2020 12 7 1934 PMC7409089 32708897
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A Review of Research on CRISPR/Cas9 Gene Editing Technology in Disease Treatment
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“For viral infections, CRISPR/Cas9 can be used to cut viral genomes and inhibit viral replication, such as in the case of HIV and Hepatitis B[7].”
- Authors
- 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.
- Affiliations
- Shanghai Medical College of Fudan University (CN)
- Fudan University (CN)
- Corresponding author
- Ke, Ai-Wu
- Venue
- Molecular Cancer
- Published
- 2022-02-21
- Volume
- 21
- Issue
- 1
- Pages
- 57-57
- ISSN
- 1476-4598 (ISSN-L)
- PMC
- PMC8862238
- OpenAlex
- W4213070361
- Cited by
- 478
- References
- 37
- Citations by year
- 2026: 78 · 2025: 182 · 2024: 128 · 2023: 78 · 2022: 12
- FWCI
- 10.44
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Molecular Cancer (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- National Natural Science Foundation of China (81672825, 81602513)
- Natural Science Foundation of Shanghai (21ZR1412200)
- Chen Guang Program of Shanghai Municipal Education Commission (CJB)
- Young Elite Scientists Sponsorship Program by CAST (2019QNRC001)
- the Clinical Research Plan of SHDC (SHDC2020CR2067B)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Chromatin Remodeling and Cancer
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Keywords
- CRISPR/Cas9, genome editing, cancer, CRISPR screen, carcinogenesis, metastasis, drug resistance, gene delivery vectors, adoptive T cell therapy, cancer genomics
- MeSH terms
- Gene Editing, Animals, Humans, Neoplasms, Oncogenes, Genomics, CRISPR-Cas Systems
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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