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Genome editing of immune cells using CRISPR/Cas9

김찬혁|김세기|Cedric Hupperetz|Seongjoon Lim
생화학분자생물학회 (Korean Society Of Biochemistry And Molecular Biology)|2021.01|BMB Reports|제54권 제1호|pp. 59-69

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키워드

Genome editingCRISPR/Cas9Engineered T cellsGenome- wide screeningHSPC engineering
주제분류화학

초록

The ability to read, write, and edit genomic information in livingorganisms can have a profound impact on research, health,economic, and environmental issues. The CRISPR/Cas system,recently discovered as an adaptive immune system in prokaryotes,has revolutionized the ease and throughput of genomeediting in mammalian cells and has proved itself indispensableto the engineering of immune cells and identification of novelimmune mechanisms. In this review, we summarize the CRISPR/Cas9 system and the history of its discovery and optimization. We then focus on engineering T cells and other types ofimmune cells, with emphasis on therapeutic applications. Last,we describe the different modifications of Cas9 and theirrecent applications in the genome-wide screening of immunecells.

Abstract

The ability to read, write, and edit genomic information in livingorganisms can have a profound impact on research, health,economic, and environmental issues. The CRISPR/Cas system,recently discovered as an adaptive immune system in prokaryotes,has revolutionized the ease and throughput of genomeediting in mammalian cells and has proved itself indispensableto the engineering of immune cells and identification of novelimmune mechanisms. In this review, we summarize the CRISPR/Cas9 system and the history of its discovery and optimization. We then focus on engineering T cells and other types ofimmune cells, with emphasis on therapeutic applications. Last,we describe the different modifications of Cas9 and theirrecent applications in the genome-wide screening of immunecells.

참고문헌 (107)

  1. Mardis ER, "DNA sequencing technologies : 2006-2016", Nat Protoc, 12, pp.213~218 (2017)
  2. Zhang W, "Synthetic genomes", Annu Rev Biochem, 89, pp.77~101 (2020)
  3. Pickar-Oliver A, "The next generation of CRISPR-Cas technologies and applications", Nat Rev Mol Cell Biol, 20, pp.490~507 (2019)
  4. Sander JD, "CRISPR-Cas systems for editing, regulating and targeting genomes", Nat Biotechnol, 32, pp.347~355 (2014)
  5. Anzalone AV, "Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors", Nat Biotechnol, 38, pp.824~844 (2020)
  6. Tong S, "Engineered materials for in vivo delivery of genome-editing machinery", Nat Rev Mater, 4, pp.726~737 (2019)
  7. Ishino Y, "Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme convertsion in Escherichia coli, and identification of the gene product", J Bacteriol, 169, pp.5429~5433 (1987)
  8. Jansen R, "Identification of genes that are associated with DNA repeats in prokaryotes", Mol Microbiol, 43, pp.1565~1575 (2002)
  9. Mojica FJ, "Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements", J Mol Evol, 60, pp.174~182 (2005)
  10. Barrangou R, "CRISPR provides acquired resistance against viruses in prokaryotes", Science, 315, pp.1709~1712 (2007)
  11. Makarova KS, "Evolutionary classification of CRISPR-Cas systems : a burst of class 2and derived variants", Nat Rev Microbiol, 18, pp.67~83 (2020)
  12. Jinek M, "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity", Science, 337, pp.816~821 (2012)
  13. Mojica FJM, "Short motif sequences determine the targets of the prokaryotic CRISPR defence system", Microbiology(Reading), 155, pp.733~740 (2009)
  14. Wang H, "CRISPR", Cas9 in genome editing and beyond, 2016, pp.85, 227~264
  15. Ren J, "Advancing chimeric antigen receptor T cell therapy with CRISPR", Cas9, 2017, pp.8, 634~643
  16. Kim S, "Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins", Genome Res, 24, pp.1012~1019 (2014)
  17. Hendel A, "Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells", Nat Biotechnol, 33, pp.985~989 (2015)
  18. Hornung V, "Intracellular DNA recognition", Nat Rev Immunol, 10, pp.123~130 (2010)
  19. Geering B, "Synthetic immunology : modulating the human immune system", Trends Biotechnol, 33, pp.65~79 (2015)
  20. Wei SC, "Fundamental mechanisms of immune checkpoint blockade therapy", Cancer Discov, 8, pp.1069~1086 (2018)
  21. Klinger M, "Harnessing T cells to fight cancer with BiTE(R)antibody constructs--past developments and future directions", Immunol Rev, 270, pp.193~208 (2016)
  22. Guedan S, "Emerging cellular therapies for cancer", Annu Rev Immunol, 37, pp.145~171 (2019)
  23. Schuster SJ, "Chimeric antigen receptor T cells in refractory B-cell lymphomas", N Engl J Med, 377, pp.2545~2554 (2017)
  24. Neelapu SS, "Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma", N Engl J Med, 377, pp.2531~2544 (2017)
  25. Maude SL, "Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia", N Engl J Med, 378, pp.439~448 (2018)
  26. Blank CU, "Defining ‘T cell exhaustion’", Nat Rev Immunol, 19, pp.665~674 (2019)
  27. Wherry EJ, "Molecular and cellular insights into T cell exhaustion", Nat Rev Immunol, 15, pp.486~499 (2015)
  28. Choi BD, "CRISPR-Cas9disruption of PD-1 enhances activity of universal EGFRvIII CAR T cells in a preclinical model of human glioblastoma", J Immunother Cancer, 7, pp.304~ (2019)
  29. Hu W, "CRISPR", Cas9-mediated PD-1 disruption enhances human mesothelin-targeted CAR T cell effector functions, 2019, pp.68, 365~377
  30. Rupp LJ, "CRISPR", Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells, 2017, pp.7, 737~
  31. Su S, "CRISPR-Cas9 mediated efficient PD-1 disruption on human primary T cells from cancer patients", Sci Rep, 6, pp.20070~ (2016)
  32. Stadtmauer EA, "CRISPR-engineered T cells in patients with refractory cancer", Science, 367, pp.eaba7365~ (2020)
  33. Yang L, "TGF-beta and immune cells : an important regulatory axis in the tumor microenvironment and progression", Trends Immunol, 31, pp.220~227 (2010)
  34. Tang N, "TGF-beta inhibition via CRISPR promotes the long-term efficacy of CAR T cells against solid tumors", JCI Insight, 5, pp.e133977~ (2020)
  35. Yamada A, "Dual role of Fas", FasL-mediated signal in peripheral immune tolerance, 2017, pp.8, 403~
  36. Ren J, "A versatile system for rapid multiplex genome-edited CAR T cell generation", Oncotarget, 8, pp.17002~17011 (2017)
  37. Riese MJ, "Diacylglycerol kinases(DGKs) : novel targets for improving T cell activity in cancer", Front Cell Dev Biol, 4, pp.108~ (2016)
  38. Jung IY, "CRISPR", Cas9-mediated knockout of DGK improves antitumor activities of human T cells, 2018, pp.78, 4692~4703
  39. Norelli M, "Monocytederived IL-1 and IL-6 are differentially required for cytokine-release syndrome and neurotoxicity due to CAR T cells", Nat Med, 24, pp.739~748 (2018)
  40. Teachey DT, "Identification of predictive biomarkers for cytokine release syndrome after chimeric antigen receptor T-cell therapy for acute lymphoblastic leukemia", Cancer Discov, 6, pp.664~679 (2016)
  41. Sterner RM, "GM-CSF inhibition reduces cytokine release syndrome and neuroinflammation but enhances CAR-T cell function in xenografts", Blood, 133, pp.697~709 (2019)
  42. Alcantara M, "CAR T-cells for T-cell malignancies : challenges in distinguishing between therapeutic, normal, and neoplastic T-cells", Leukemia, 32, pp.2307~2315 (2018)
  43. Gomes-Silva D, "CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies", Blood, 130, pp.285~296 (2017)
  44. Ren J, "Multiplex genome editing to generate universal CAR T cells resistant to PD1 inhibition", Clin Cancer Res, 23, pp.2255~2266 (2017)
  45. Morton LT, "Simultaneous deletion of endogenous TCRalphabeta for TCR gene therapy creates an improved and safe cellular therapeutic", Mol Ther, 28, pp.64~74 (2020)
  46. Maher J, "Human T-lymphocyte cytotoxicity and proliferation directed by a single chimeric TCRzeta", CD28 receptor, 2002, pp.20, 70~75
  47. Milone MC, "Chimeric receptors containing CD137 signal transduction domains mediate enhanced survival of T cells and increased antileukemic efficacy in vivo", Mol Ther, 17, pp.1453~1464 (2009)
  48. Uren AG, "Retroviral insertional mutagenesis : past, present and future", Oncogene, 24, pp.7656~7672 (2005)
  49. Niederer HA, "Integration site and clonal expansion in human chronic retroviral infection and gene therapy", Viruses, 6, pp.4140~4164 (2014)
  50. Woods NB, "Lentiviral vector transduction of NOD", SCID repopulating cells results in multiple vector integrations per transduced cell : risk of insertional mutagenesis, 2003, pp.101, 1284~1289
  51. Eyquem J, "Targeting a CAR to the TRAC locus with CRISPR", Cas9 enhances tumour rejection, 2017, pp.543, 113~117
  52. Wright JF, "Manufacturing and characterizing AAVbased vectors for use in clinical studies", Gene Ther, 15, pp.840~848 (2008)
  53. Roth TL, "Reprogramming human T cell function and specificity with non-viral genome targeting", Nature, 559, pp.405~409 (2018)
  54. Nguyen DN, "Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency", Nat Biotechnol, 38, pp.44~49 (2020)
  55. Roth TL, "Pooled Knockin Targeting for Genome Engineering of Cellular Immunotherapies", Cell, 181, pp.728e721~744e721 (2020)
  56. Tothova Z, "Multiplex CRISPR", Cas9-based genome editing in human hematopoietic stem cells models clonal hematopoiesis and myeloid neoplasia, 2017, pp.21, 547e8~555e8
  57. Wagenblast E, "Functional profiling of single CRISPR", Cas9-edited human long-term hematopoietic stem cells, 2019, pp.10, 4730~
  58. Cavazzana M, "Gene therapy for beta-hemoglobinopathies", Mol Ther, 25, pp.1142~1154 (2017)
  59. Dever DP, "CRISPR", Cas9beta-globin gene targeting in human haematopoietic stem cells, 2016, pp.539, 384~389
  60. Wu Y, "Highly efficient therapeutic gene editing of human hematopoietic stem cells", Nat Med, 25, pp.776~783 (2019)
  61. Zeng J, "Therapeutic base editing of human hematopoietic stem cells", Nat Med, 26, pp.535~541 (2020)
  62. Traxler EA, "A genome-editing strategy to treat beta-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition", Nat Med, 22, pp.987~990 (2016)
  63. Weber L, "Editing a gammaglobin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype", Sci Adv, 6, pp.eaay9392~ (2020)
  64. Hacein-Bey-Abina S, "Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1", J Clin Invest, 118, pp.3132~3142 (2008)
  65. Woods NB, "Gene therapy : therapeutic gene causing lymphoma", Nature, 440, pp.1123~ (2006)
  66. Pavel-Dinu M, "Gene correction for SCID-X1 in long-term hematopoietic stem cells", Nat Commun, 10, pp.1634~ (2019)
  67. Holt N, "Human hematopoietic stem", progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo, 2010, pp.28, 839~847
  68. Shi B, "TALEN-mediated knockout of CCR5 confers protection against infection of human immunodeficiency virus", J Acquir Immune Defic Syndr, 74, pp.229~241 (2017)
  69. Xu L, "CRISPR", Cas9-mediated CCR5 ablation in human hematopoietic stem, Mol Ther, pp.2017, 25[DOI]
  70. Xu L, "CRISPR-edited stem cells in a patient with HIV and acute lymphocytic leukemia", N Engl J Med, 381, pp.1240~1247 (2019)
  71. Hartweger H, "HIVspecific humoral immune responses by CRISPR", Cas9-edited B cells, 2019, pp.216, 1301~1310
  72. Moffett HF, "B cells engineered to express pathogen-specific antibodies protect against infection", Sci Immunol, 4, pp.eaax0644~ (2019)
  73. McManus MT, "Gene silencing in mammals by small interfering RNAs", Nat Rev Genet, 3, pp.737~747 (2002)
  74. Jackson AL, "Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application", Nat Rev Drug Discov, 9, pp.57~67 (2010)
  75. Munoz DM, "CRISPR screens provide a comprehensive assessment of cancer vulnerabilities but generate false-positive hits for highly amplified genomic regions", Cancer Discov, 6, pp.900~913 (2016)
  76. Tian X, "CRISPR", Cas9-an evolving biological tool kit for cancer biology and oncology, 2019, pp.3, 8~
  77. Wang T, "Genetic screens in human cells using the CRISPR-Cas9system", Science, 343, pp.80~84 (2014)
  78. Mali P, "CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering", Nat Biotechnol, 31, pp.833~838 (2013)
  79. Perez-Pinera P, "RNA-guided gene activation by CRISPR-Cas9-based transcription factors", Nat Methods, 10, pp.973~976 (2013)
  80. Konermann S, "Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex", Nature, 517, pp.583~588 (2015)
  81. Zalatan JG, "Engineering complex synthetic transcriptional programs with CRISPR RNA scaffolds", Cell, 160, pp.339~350 (2015)
  82. Gilbert LA, "CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes", Cell, 154, pp.442~451 (2013)
  83. McDade JR, "Practical considerations for using pooled lentiviral CRISPR libraries", Curr Protoc Mol Biol, 115, pp.31.5.1~31.5.13 (2016)
  84. Joung J, "Genome-scale CRISPR-Cas9 knockout and transcriptional activation screening", Nat Protoc, 12, pp.828~863 (2017)
  85. Park RJ, "A genomewide CRISPR screen identifies a restricted set of HIV host dependency factors", Nat Genet, 49, pp.193~203 (2017)
  86. Ait-Ammar A, "Current status of latency reversing agents facing the heterogeneity of HIV-1 cellular and tissue reservoirs", Front Microbiol, 10, pp.3060~ (2019)
  87. Rathore A, "CRISPR-based gene knockout screens reveal deubiquitinases involved in HIV-1 latency in two Jurkat cell models", Sci Rep, 10, pp.5350~ (2020)
  88. Manganaro L, "Tumor suppressor cylindromatosis(CYLD). controls HIV transcription in an NF-kappaB-dependent manner", J Virol, 88, pp.7528~7540 (2014)
  89. Pan D, "A major chromatin regulator determines resistance of tumor cells to T cell-mediated killing", Science, 359, pp.770~775 (2018)
  90. Kadoch C, "Mammalian SWI", SNF chromatin remodeling complexes and cancer : Mechanistic insights gained from human genomics, 2015, pp.1, e1500447~
  91. Singh N, "Impaired death receptor signaling in leukemia causes antigen-independent resistance by inducing CAR T-cell dysfunction", Cancer Discov, 10, pp.552~567 (2020)
  92. Cortez JT, "CRISPR screen in regulatory T cells reveals modulators of Foxp3", Nature, 582, pp.416~420 (2020)
  93. Henriksson J, "CRISPR screening in single cells", Methods Mol Biol, 1979, pp.395~406 (2019)
  94. Klein AM, "Droplet barcoding for single-cell transcriptomics applied to embryonic stem cells", Cell, 161, pp.1187~1201 (2015)
  95. Macosko EZ, "Highly parallel genome-wide expression profiling of individual cells using nanoliter droplets", Cell, 161, pp.1202~1214 (2015)
  96. Dixit A, "Perturb-Seq : dissecting molecular circuits with scalable single-cell RNA profiling of pooled genetic screens", Cell, 167, pp.1853e17~1866e17 (2016)
  97. Xie S, "Multiplexed engineering and analysis of combinatorial enhancer activity in single cells", Mol Cell, 66, pp.285e5~299e5 (2017)
  98. Jaitin DA, "Dissecting immune circuits by linking CRISPR-pooled screens with single-cell RNA-Seq", Cell, 167, pp.1883e15~1896e15 (2016)
  99. Datlinger P, "Pooled CRISPR screening with single-cell transcriptome readout", Nat Methods, 14, pp.297~301 (2017)
  100. Adamson B, "A multiplexed single-cell CRISPR screening platform enables systematic dissection of the unfolded protein response", Cell, 167, pp.1867e21~1882e21 (2016)
  101. McFaline-Figueroa JL, "A pooled single-cell genetic screen identifies regulatory checkpoints in the continuum of the epithelial-to-mesenchymal transition", Nat Genet, 51, pp.1389~1398 (2019)
  102. Chopin M, "Transcription factor PU. 1 promotes conventional dendritic cell identity and function via induction of transcriptional regulator DC-SCRIPT", Immunity, 50, pp.77.e5~90.e5 (2019)
  103. Haapaniemi E, "CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response", Nat Med, 24, pp.927~930 (2018)
  104. Cullot G, "CRISPR-Cas9genome editing induces megabase-scale chromosomal truncations", Nat Commun, 10, pp.1136~ (2019)
  105. Webber BR, "Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors", Nature Communications, 10, pp.5222~ (2019)
  106. Strecker J, "RNA-guided DNA insertion with CRISPR-associated transposases", Science, 365, pp.48~53 (2019)
  107. Klompe SE, "Transposon-encoded CRISPR-Cas systems direct RNA-guided DNA integration", Nature, 571, pp.219~225 (2019)

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