基于CRISPR/Cas9的基因编辑技术及其在肿瘤治疗中的应用

doi:10.3760/cma.j.cn371439-20220408-00106

摘要/Abstract

摘要：

CRISPR/Cas9及其衍生的基因编辑技术碱基编辑器和先导编辑器可对目标基因组进行精准编辑,已广泛应用于肿瘤的治疗,在肿瘤免疫疗法、治疗人乳头瘤病毒感染、构建高效溶瘤病毒等方面取得了显著成果,为肿瘤治疗提供了新手段。

关键词: 肿瘤, 基因编辑, 免疫疗法, 溶瘤病毒疗法, CRISPR/Cas9基因编辑技术

Abstract:

Gene editing technology CRISPR/Cas9 and its derivative editing technologies including base editor and prime editor can precisely edit the target genome sequences, having been widely used in tumor therapy and achieved remarkable clinical results in tumor immunotherapy, human papilloma virus infection treatment and oncolytic virotherapy, providing a new means for tumor therapy.

Key words: Neoplasms, Gene editing, Immunotherapy, Oncolytic virotherapy, CRISPR/Cas9 genome editing technique

张子悦, 郑斯豪, 高彦君, 姚颐, 宋启斌. 基于CRISPR/Cas9的基因编辑技术及其在肿瘤治疗中的应用[J]. 国际肿瘤学杂志, 2022, 49(9): 546-549.

Zhang Ziyue, Zheng Sihao, Gao Yanjun, Yao Yi, Song Qibin. CRISPR/Cas9 genome editing technology and its applications in tumor therapy[J]. Journal of International Oncology, 2022, 49(9): 546-549.

参考文献 36

[1]	Balon K, Sheriff A, Jacków J, et al. Targeting cancer with CRISPR/Cas9-based therapy[J]. Int J Mol Sci, 2022, 23(1): 573. DOI: 10.3390/ijms23010573. doi: 10.3390/ijms23010573
[2]	Jiang C, Meng L, Yang B, et al. Application of CRISPR/Cas9 gene editing technique in the study of cancer treatment[J]. Clin Genet, 2020, 97(1): 73-88. DOI: 10.1111/cge.13589. doi: 10.1111/cge.13589 pmid: 31231788
[3]	Gupta D, Bhattacharjee O, Mandal D, et al. CRISPR-Cas9 system: a new-fangled dawn in gene editing[J]. Life Sci, 2019, 232: 116636. DOI: 10.1016/j.lfs.2019.116636. doi: 10.1016/j.lfs.2019.116636
[4]	Miller SM, Wang T, Randolph PB, et al. Continuous evolution of SpCas9 variants compatible with non-G PAMs[J]. Nat Biotechnol, 2020, 38(4): 471-481. DOI: 10.1038/s41587-020-0412-8. doi: 10.1038/s41587-020-0412-8 pmid: 32042170
[5]	Walton RT, Christie KA, Whittaker MN, et al. Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants[J]. Science, 2020, 368(6488): 290-296. DOI: 10.1126/science.aba8853. doi: 10.1126/science.aba8853 pmid: 32217751
[6]	Leibowitz ML, Papathanasiou S, Doerfler PA, et al. Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing[J]. Nat Genet, 2021, 53(6): 895-905. DOI: 10.1038/s41588-021-00838-7. doi: 10.1038/s41588-021-00838-7 pmid: 33846636
[7]	Haapaniemi E, Botla S, Persson J, et al. CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response[J]. Nat Med, 2018, 24(7): 927-930. DOI: 10.1038/s41591-018-0049-z. doi: 10.1038/s41591-018-0049-z pmid: 29892067
[8]	Dai X, Blancafort P, Wang P, et al. Innovative precision gene-editing tools in personalized cancer medicine[J]. Adv Sci (Weinh), 2020, 7(12): 1902552. DOI: 10.1002/advs.201902552. doi: 10.1002/advs.201902552
[9]	Zhang X, Zhu B, Chen L, et al. Dual base editor catalyzes both cytosine and adenine base conversions in human cells[J]. Nat Biotechnol, 2020, 38(7): 856-860. DOI: 10.1038/s41587-020-0527-y. doi: 10.1038/s41587-020-0527-y pmid: 32483363
[10]	Grünewald J, Zhou R, Lareau CA, et al. A dual-deaminase CRISPR base editor enables concurrent adenine and cytosine editing[J]. Nat Biotechnol, 2020, 38(7): 861-864. DOI: 10.1038/s41587-020-0535-y. doi: 10.1038/s41587-020-0535-y pmid: 32483364
[11]	Xin H, Wan T, Ping Y. Off-targeting of base editors: BE3 but not ABE induces substantial off-target single nucleotide variants[J]. Signal Transduct Target Ther, 2019, 4: 9. DOI: 10.1038/s41392-019-0044-y. doi: 10.1038/s41392-019-0044-y
[12]	Zuo E, Sun Y, Wei W, et al. Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos[J]. Science, 2019, 364(6437): 289-292. DOI: 10.1126/science.aav9973. doi: 10.1126/science.aav9973 pmid: 30819928
[13]	da Costa BL, Levi SR, Eulau E, et al. Prime editing for inherited retinal diseases[J]. Front Genome Ed, 2021, 3: 775330. DOI: 10.3389/fgeed.2021.775330. doi: 10.3389/fgeed.2021.775330
[14]	Anzalone AV, Randolph PB, Davis JR, et al. Search-and-replace genome editing without double-strand breaks or donor DNA[J]. Nature, 2019, 576(7785): 149-157. DOI: 10.1038/s41586-019-1711-4. doi: 10.1038/s41586-019-1711-4
[15]	Kagoya Y, Guo T, Yeung B, et al. Genetic ablation of HLA class Ⅰ, class Ⅱ, and the t-cell receptor enables allogeneic T cells to be used for adoptive t-cell therapy[J]. Cancer Immunol Res, 2020, 8(7): 926-936. DOI: 10.1158/2326-6066.CIR-18-0508. doi: 10.1158/2326-6066.CIR-18-0508 pmid: 32321775
[16]	Zhao Z, Li C, Tong F, et al. Review of applications of CRISPR-Cas9 gene-editing technology in cancer research[J]. Biol Proced Online, 2021, 23(1): 14. DOI: 10.1186/s12575-021-00151-x. doi: 10.1186/s12575-021-00151-x
[17]	Gao Q, Dong X, Xu Q, et al. Therapeutic potential of CRISPR/Cas9 gene editing in engineered T-cell therapy[J]. Cancer Med, 2019, 8(9): 4254-4264. DOI: 10.1002/cam4.2257. doi: 10.1002/cam4.2257
[18]	Choi BD, Yu X, Castano AP, et al. CRISPR-Cas9 disruption of PD-1 enhances activity of Universal EGFRvⅢ CAR T cells in a preclinical model of human glioblastoma[J]. J Immunother Cancer, 2019, 7(1): 304. DOI: 10.1186/s40425-019-0806-7. doi: 10.1186/s40425-019-0806-7
[19]	Morimoto T, Nakazawa T, Matsuda R, et al. CRISPR-Cas9-mediated TIM3 knockout in human natural killer cells enhances growth inhibitory effects on human glioma cells[J]. Int J Mol Sci, 2021, 22(7): 3489. DOI: 10.3390/ijms22073489. doi: 10.3390/ijms22073489
[20]	Jung IY, Kim YY, Yu HS, et al. CRISPR/Cas9-Mediated knockout of DGK improves antitumor activities of human T cells[J]. Cancer Res, 2018, 78(16): 4692-4703. DOI: 10.1158/0008-5472.CAN-18-0030. doi: 10.1158/0008-5472.CAN-18-0030
[21]	Tang N, Cheng C, Zhang X, et al. TGF-β inhibition via CRISPR promotes the long-term efficacy of CAR T cells against solid tumors[J]. JCI Insight, 2020, 5(4): e133977. DOI: 10.1172/jci.insight.133977. doi: 10.1172/jci.insight.133977
[22]	Wang Z, Li N, Feng K, et al. Phase Ⅰ study of CAR-T cells with PD-1 and TCR disruption in mesothelin-positive solid tumors[J]. Cell Mol Immunol, 2021, 18(9): 2188-2198. DOI: 10.1038/s41423-021-00749-x. doi: 10.1038/s41423-021-00749-x
[23]	Webber BR, Lonetree CL, Kluesner MG, et al. Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors[J]. Nat Commun, 2019, 10(1): 5222. DOI: 10.1038/s41467-019-13007-6. doi: 10.1038/s41467-019-13007-6
[24]	Ou X, Ma Q, Yin W, et al. CRISPR/Cas9 gene-editing in cancer immunotherapy: promoting the present revolution in cancer therapy and exploring more[J]. Front Cell Dev Biol, 2021, 9: 674467. DOI: 10.3389/fcell.2021.674467. doi: 10.3389/fcell.2021.674467
[25]	Morton LT, Reijmers RM, Wouters AK, et al. Simultaneous deletion of endogenous TCRαβ for TCR gene therapy creates an improved and safe cellular therapeutic[J]. Mol Ther, 2020, 28(1): 64-74. DOI: 10.1016/j.ymthe.2019.10.001. doi: S1525-0016(19)30455-1 pmid: 31636040
[26]	Stadtmauer EA, Fraietta JA, Davis MM, et al. CRISPR-engineered T cells in patients with refractory cancer[J]. Science, 2020, 367(6481): eaba7365. DOI: 10.1126/science.aba7365. doi: 10.1126/science.aba7365
[27]	Guo X, Jiang H, Shi B, et al. Disruption of PD-1 enhanced the anti-tumor activity of chimeric antigen receptor T cells against hepatocellular carcinoma[J]. Front Pharmacol, 2018, 9: 1118. DOI: 10.3389/fphar.2018.01118. doi: 10.3389/fphar.2018.01118 pmid: 30327605
[28]	He XY, Ren XH, Peng Y, et al. Aptamer/peptide-functionalized genome-editing system for effective immune restoration through reversal of PD-L1-Mediated cancer immunosuppression[J]. Adv Mater, 2020, 32(17): e2000208. DOI: 10.1002/adma.202000208. doi: 10.1002/adma.202000208
[29]	Lu Y, Xue J, Deng T, et al. Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer[J]. Nat Med, 2020, 26(5): 732-740. DOI: 10.1038/s41591-020-0840-5. doi: 10.1038/s41591-020-0840-5 pmid: 32341578
[30]	Jubair L, Fallaha S, McMillan NAJ. Systemic delivery of CRISPR/Cas9 targeting HPV oncogenes is effective at eliminating established tumors[J]. Mol Ther, 2019, 27(12): 2091-2099. DOI: 10.1016/j.ymthe.2019.08.012. doi: S1525-0016(19)30395-8 pmid: 31537455
[31]	Zhang H, Qin C, An C, et al. Application of the CRISPR/Cas9-based gene editing technique in basic research, diagnosis, and therapy of cancer[J]. Mol Cancer, 2021, 20(1): 126. DOI: 10.1186/s12943-021-01431-6. doi: 10.1186/s12943-021-01431-6 pmid: 34598686
[32]	Xiong J, Tan S, Yu L, et al. E7-targeted nanotherapeutics for key HPV afflicted cervical lesions by employing CRISPR/Cas9 and poly (beta-amino ester)[J]. Int J Nanomedicine, 2021, 16: 7609-7622. DOI: 10.2147/IJN.S335277. doi: 10.2147/IJN.S335277
[33]	Chen M, Mao A, Xu M, et al. CRISPR-Cas 9 for cancer therapy: opportunities and challenges[J]. Cancer Lett, 2019, 447: 48-55. DOI: 10.1016/j.canlet.2019.01.017. doi: 10.1016/j.canlet.2019.01.017
[34]	Azangou-Khyavy M, Ghasemi M, Khanali J, et al. CRISPR/Cas: from tumor gene editing to T Cell-Based immunotherapy of cancer[J]. Front Immunol, 2020, 11: 2062. DOI: 10.3389/fimmu.2020.02062. doi: 10.3389/fimmu.2020.02062 pmid: 33117331
[35]	Ebrahimi S, Makvandi M, Abbasi S, et al. Developing oncolytic Herpes simplex virus type 1 through UL39 knockout by CRISPR-Cas9[J]. Iran J Basic Med Sci, 2020, 23(7): 937-944. DOI: 10.22038/ijbms.2020.43864.10286. doi: 10.22038/ijbms.2020.43864.10286 pmid: 32774817
[36]	Cai L, Hu H, Duan H, et al. The construction of a new oncolytic herpes simplex virus expressing murine interleukin-15 with gene-editing technology[J]. J Med Virol, 2020, 92(12): 3617-3627. DOI: 10.1002/jmv.25691. doi: 10.1002/jmv.25691