Advances in CRISPR-Cas9 Technology for Tumor Therapy
DOI:
https://doi.org/10.61173/bk6wsg49Keywords:
Gene editing, CRISPR-Cas9, Tumor TherapyAbstract
Genome editing technology is an emerging toolbox that can specifically target genes. At present, the existing gene editing technologies include ZFN, TALEN, and CRISPR system editing technology with clusters of regularly spaced short palindromic repeats. However, ZFN and TALEN are no longer the preferred tools for gene editing due to some of their problems, and their complex protein design, high cost, and high difficulty are the main reasons that limit their use. The CRISPR system, on the other hand, is able to achieve specific binding by base complementary pairing of sgRNA to the target sequence. Traditional cancer treatment methods have limited efficacy and high recurrence rates, and the disease can be fundamentally treated by inactivating oncogenes or activating tumor suppressor genes through gene editing technology and then changing downstream signaling pathways for cancer treatment. With CRISPR-Cas9 technology, it is possible to have gene knockouts, insertions, and mutations. Here, we will investigate and explore the feasibility of the CRISPR system for tumor treatment, including the following three topics: oncogene knockout, augmentation of tumor suppressor gene expression, and enhancement of engineered T cell viability for effective tumor Through these methods, the CRISPR system is of great help and promising tumor therapy.
References
[1] DREIER B, FULLER R P, SEGAL D J, et al. Development of zinc finger domains for recognition of the 5'-CNN-3' family DNA sequences and their use in the construction of artificial transcription factors[J]. Journal of Biological Chemistry, 2005, 280( 42) 35588-35597. DOI: 10. 1074 /jbc. M506654200.
[2] TESSON L, USAL C, MENORET S, et al. Knockout rats generated by embryo microinjection of TALENs[J]. Nature Biotechnology, 2011, 29( 8) : 695-696. DOI: 10. 1038 /nbt. 1940.
[3] CHEN H F, CHOI J, BAILEY S. Cut site selection by the two nuclease domains of the Cas9 RNA-guided endonuclease[J]. Journal of Biological Chemistry, 2014, 289( 19) : 13284-13294. DOI: 10. 1074 /jbc. M113. 539726. [11] SASAKI H, YOSHIDA K, HOZUMI A, et al. CRISPR/Cas9-mediated gene knockout in the ascidian Ciona intestinalis[J]. Development Growth Differentiation, 2014, 56( 7) : 499-510. DOI: 10. 1111 /dgd. 12149.
[4] KLUG A, RHODES D. Zinc fingers: a novel protein fold for nucleic acid recognition[J]. Cold Spring Harbor Symposia on Quantitative Biology, 1987, 52: 473 - 482. [14] KIM Y G, CHA J, CHANDRASEGARAN S. Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain[J]. Proceedings of the National Academy of Sciences, 1996, 93( 3) : 1156 - 1160.
[5] WOOD A J, LO T W, ZEITLER B, et al. Targeted genome editing across species using ZFNs and TALENs[J]. Science, 2011, 333( 6040) : 307. [22] MILLER J C, TAN S, QIAO G, et al. A TALE nuclease architecture for efficient genome editing[J]. Nature Biotechnology, 2011, 29( 2) : 143 - 148.
[6] JOUNG J K, SANDER J D. TALENs: a widely applicable technology for targeted genome editing[J]. Nature Reviews Molecular Cell Biology, 2013, 14( 1) : 49 - 55.
[7] JINEK M, CHYLINSKI K, FONFARA I, et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity[J]. Science, 2012, 337 ( 6096 ) : 816 -821.
[8] NORAIS C, MOISAN A, GASPIN C, et al. Diversity of CRISPR systems in the euryarchaeal pyrococcales[J]. RNA Biology, 2013, 10( 5) : 659-670. DOI: 10. 4161 /rna. 23927.
[9] WEI Y Z, CHESNE M T, TERNS R M, et al. Sequences spanning the leader-repeat junction mediate CRISPR adaptation to phage in Streptococcus thermophilus[J]. Nucleic Acids Research, 2015, 43( 3) : 1749-1758. DOI: 10. 1093 /nar / gku1407.
[10] NORAIS C, MOISAN A, GASPIN C, et al. Diversity of CRISPR systems in the euryarchaeal pyrococcales[J]. RNA Biology, 2013, 10( 5) : 659-670. DOI: 10. 4161 /rna. 23927.
[11] OH J-H, VAN PIJKEREN J-P. CRISPR-Cas9-assisted recombineering in Lactobacillus reuteri[J]. Nucleic Acids Research, 2014, 42( 17) : 1-11. DOI:10.1093/nar/gku623
[12] MALI P, ESVELT K M, CHURCH G M. Cas9 as a versatile tool for engineering biology[J]. Nature Methods, 2013, 10( 10) :957-963. DOI: 10. 1038 /NMETH. 2649.
[13] DELTCHEVA E, CHYLINSKI K, SHARMA C M, et al. CRISPR RNA maturation by trans-encoded small RNA and host factorRNase III[J]. Nature, 2011, 471( 7340) : 602-607. DOI: 10. 1038 /nature09886
[14] Makarova KS, Wolf YI, Alkhnbashi OS, et al. An updated evolutionary classification of CRISPR-Cas systems. Nat Rev Microbiol, 2015, 13: 722-36
[15] Makarova KS, Zhang F, Koonin EV. SnapShot: class 1 CRISPR-Cas systems. Cell, 2017, 168: 946-946.e941
[16] Shmakov S, Smargon A, Scott D, et al. Diversity and evolution of class 2 CRISPR-Cas systems. Nat Rev Microbiol, 2017, 15: 169-82
[17] Taylor HN, Warner EE, Armbrust MJ, et al. Structural basis of type IV CRISPR RNA biogenesis by a Cas6 endoribonuclease. RNA Biol, 2019, 16: 1438-47
[18] CONG L, COX D, LIN SL, BARRETTO R, HABIB N, HSU PD, WU XB, JIANG WY, MARRAFFINI LA, ZHANG F. Multiplex genome engineering using CRISPR/Cas systems[J]. Dean&Francis Science, 2013, 339(6121): 819-823.
[19] JINEK M, CHYLINSKI K, FONFARA I, HAUER M, DOUDNA JA, CHARPENTIER E. A programmable dual-RNA- guided DNA endonuclease in adaptive bacterial immunity[J]. Science, 2012, 337(6096): 816-821.
[20] Arzate-Mejia RG, Licona-Limon P, Recillas-Targa F. Genome editing during development using the CRISPRCas technology. Methods Mol Biol, 2018, 1752: 177-90.
[21] Yosef I, Goren MG, Qimron U. Proteins and DNA elements essential for the CRISPR adaptation process in Escherichia coli. Nucleic Acids Res, 2012, 40: 5569-76.
[22] BRINKMAN EK, CHEN T, de HAAS M, HOLLAND HA, AKHTAR W, van STEENSEL B. Kinetics and fidelity of the repair of Cas9-induced double-strand DNA breaks[J]. Molecular Cell, 2018, 70(5): 801-813.e6.
[23] KOMOR AC, BADRAN AH, LIU DR. CRISPR-based technologies for the manipulation of eukaryotic genomes[J]. Cell, 2017, 168(1/2): 20-36.
[24] KOMOR AC, KIM YB, PACKER MS, ZURIS JA, LIU DR. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage[J]. Nature, 2016, 533(7603): 420-424.
[25] Di NOIA J, NEUBERGER MS. Altering the pathway of immunoglobulin hypermutation by inhibiting uracil- DNA glycosylase[J]. Nature, 2002, 419(6902): 43-48.
[26] GAUDELLI NM, KOMOR AC, REES HA, PACKER MS, BADRAN AH, BRYSON DI, LIU DR.a)Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage[J]. Nature, 2017, 551(7681): 464-471.
[27] ANZALONE A V, RANDOLPH P B, DAVIS J R, et al. Searchand-replace genome editing without double-strand breaks or donor DNA[J]. Nature, 2019, 576(7785): 149-157.
[28] LI Y, LI W, LI J. The CRISPR-Cas9 revolution continues: From base editing to prime editing in plant science[J]. Journal of Genetics and Genomics, 2021, 48(8): 661-670.
[29] Gilbert LA, Larson MH, Morsut L, et al. CRISPRmediated modular RNA-guided regulation of transcription in eukaryotes. Cell, 2013, 154: 442-51
[30] Konermann S, Brigham MD, Trevino AE, et al. Genomescale transcriptional activation by an engineered CRISPRCas9 complex. Nature, 2015, 517: 583-8
[31] Fang N, Gu T, Wang Y, et al. Expression of PTEN-long mediated by CRISPR-Cas9 can repress U87 cell proliferation. J Cell Mol Med, 2017, 21,3337 ~ 3346.
[32] Chen, Z.H., Yu, Y.P., Zuo, Z.H., Nelson, J.B., Michalopoulos, G.K., Monga, S., Liu, S., Tseng, G. and Luo, J.H. (2017) Targeting genomic rearrangements in tumor cells through Cas9-mediated insertion of a suicide gene. Nat Biotechnol, 35, 543-550.
[33] Pollicino T, Cacciola I, Saffioti F, et al. Hepatitis B virus PreS /S gene variants: pathobiology and clinical implications.
[34] J Hepatol, 2014, 61, 408-417.
[35] Song J, Zhang X, Ge Q, et al. CRISPR-Cas9-mediated knockout of HBsAg inhibits proliferation and tumorigenicity
[36] of HBV-positive hepatocellular carcinoma cells. J Cell Biochem, 2018, 119, 8419 ~ 8431.
[37] Chen ZH, Yu YP, Zuo ZH, et al. Targeting genomic rearrangements in tumor cells through Cas9-mediated insertion of a suicide gene. Nat Biotechnol, 2017, 35, 543 ~ 550.
[38] Martinez-Lage M, Torres-Ruiz R, Puig-Serra P, et al. In vivo CRISPR-Cas9 targeting of fusion oncogenes for selective elimination of cancer cells. Nat Commun, 2020,11: 5060
[39] Zhen S, Lu JJ, Wang LJ, et al. In vitro and in vivo synergistic therapeutic effect of cisplatin with human papillomavirus16 E6/E7 CRISPR-Cas9 on cervical cancer cell line. Transl Oncol, 2016, 9: 498-504
[40] Li C, Mei H, Hu Y. Applications and explorations of CRISPR-Cas9 in CAR T-cell therapy. Brief Funct Genomics, 2020, 19: 175-82
[41] You L, Tong R, Li M, et al. Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research. Mol Ther Methods Clin Dev, 2019, 13, 359 ~ 370.
[42] Dong MB, Wang G, Chow RD, et al. Systematic immunotherapy target discovery using genome-scale in vivo CRISPR screens in CD8 T cells. Cell, 2019, 178, 11891204.
[43] Ye L, Park JJ, Dong MB, et al. In vivo CRISPR screening in CD8 T cells with AAV-Sleeping Beauty hybrid vectors identifies membrane targets for improving immunotherapy for glioblastoma. Nat Biotechnol, 2019, 37, 1302 ~ 1313.
[44] Fellmann C, Gowen BG, Lin PC, et al. Cornerstones of CRISPR-Cas in drug discovery and therapy. Nat Rev Drug Discov, 2017, 16, 89 ~ 100.
[45] Zanetti M. A second chance for telomerase reverse transcriptase in anticancer immunotherapy. Nat Rev Clin Oncol, 2017, 14, 115-128.
[46] Zanetti M. A second chance for telomerase reverse transcriptase in anticancer immunotherapy. Nat Rev Clin Oncol, 2017, 14, 115-128.
[47] Eyquem J, Mansilla-Soto J, Giavridis T, et al. Targeting a CAR to the TRAC locus with CRISPR-Cas9 enhances tumour rejection. Nature, 2017, 543, 113-117.
[48] Kim MY, Yu KR, Kenderian SS, et al. Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia. Cell, 2018, 173, 1439 ~ 1453.
[49] Stadtmauer EA, Fraietta JA, Davis MM, et al. CRISPR- engineered T cells in patients with refractory cancer. Science, 2020, 367, eaba7365.
[50] Lambert JM, Morris CQ. Antibody-drug conjugates( ADCs) for personalized treatment of solid tumors: a review. Adv Ther, 2017, 34, 1015 ~ 1035.
[51] Mahipal A, Grothey A. Role of biologics in first-line treatment of colorectal cancer. J Oncol Pract, 2016, 12, 1219 ~ 1228. Dean&Francis
[52] Wang G, Chow RD, Bai Z, et al. Multiplexed activation of endogenous genes by CRISPRa elicits potent antitumor immunity. Nat Immunol, 2019, 20, 1494 ~ 1505.
[53] Cheong TC, Compagno M, Chiarle R. Editing of mouse and human immunoglobulin genes by CRISPR-Cas9 system. Nat Commun, 2016, 7, 10934.
[54] Behan FM, Iorio F, Picco G, et al. Prioritization of cancer therapeutic targets using CRISPR-Cas9 screens. Nature, 2019, 568: 511-6
[55] Liu B, Diaz Arguello OA, Chen D, et al. CRISPRmediated ablation of overexpressed EGFR in combination with sunitinib significantly suppresses renal cell carcinoma proliferation. PLoS One, 2020, 15: e0232985
[56] Lee J, Choi A, Cho SY, et al. Genome-scale CRISPR screening identifies cell cycle and protein ubiquitination processes as druggable targets for erlotinib-resistant lung
[57] cancer. Mol Oncol, 2021, 15: 487-502
[58] Zhang XH, Tee LY, Wang XG, et al. Off-target effects in CRISPR-Cas9-mediated genome engineering. Mol Ther Nucleic Acids, 2015, 4: e264
[59] Sharma G, Sharma AR, Bhattacharya M, et al. CRISPRCas9: a preclinical and clinical perspective for the treatment of human diseases. Mol Ther, 2021, 29: 571-86
[60] Kleinstiver BP, Prew MS, Tsai SQ, et al. Engineered CRISPR-Cas9 nucleases with altered PAM specificities. Nature, 2015, 523: 481-5
[61] Doench JG, Fusi N, Sullender M, et al. Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9. Nat Biotechnol, 2016, 34: 184- 91
[62] Kleinstiver BP, Pattanayak V, Prew MS, et al. Highfidelity CRISPR-Cas9 nucleases with no detectable genome-wide offtarget effects. Nature, 2016, 529: 490-5
[63] Song X, Liu C, Wang N, et al. Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy. Adv Drug Deliv Rev, 2021, 168: 158-80
[64] Chen F, Alphonse M, Liu Q. Strategies for nonviral nanoparticle-based delivery of CRISPR-Cas9 therapeutics. Wiley Interdiscip Rev Nanomed Nanobiotechnol, 2020, 12: e1609
[65] Hu Z, Zhang C, Wang S, et al. Discovery and engineering of small SlugCas9 with broad targeting range and high specificity and activity. Nucleic Acids Res, 2021, 49: 4008-19
[66] Leibowitz ML, Papathanasiou S, Doerfler PA, et al. Chromothripsis as an on-target consequence of CRISPRCas9 genome editing. Nat Genet, 2021, 53: 895-905
[67] Bian S, Hou Y, Zhou X, et al. Single-cell multiomics sequencing and analyses of human colorectal cancer. Science, 2018, 362: 1060-3
[68] Zhou Y, Bian S, Zhou X, et al. Single-cell multiomics sequencing reveals prevalent genomic alterations in tumor stromal cells of human colorectal cancer. Cancer Cell, 2020, 38: 818-28.e815
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