References
1. Turtle CJ, Hanafi L-A, Berger C, et al. Immunotherapy of non-Hodgkin’s lymphoma with a defined ratio of CD8+ and CD4+ CD19-specific chimeric antigen receptor-modified T cells. Sci Transl Med . 2016;8(355):355ra116. doi:10.1126/scitranslmed.aaf8621
2. Porter DL, Levine BL, Kalos M, Bagg A, June CH. Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia. N Engl J Med . 2011;365(8):725-733. doi:10.1056/NEJMoa1103849
3. Turtle CJ, Hanafi L-A, Berger C, et al. CD19 CAR-T cells of defined CD4+:CD8+ composition in adult B cell ALL patients. J Clin Invest . 2016;126(6):2123-2138. doi:10.1172/JCI85309
4. Maude SL, Frey N, Shaw PA, et al. Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia. N Engl J Med . 2014;371(16):1507-1517. doi:10.1056/NEJMoa1407222
5. Wang Z, Wu Z, Liu Y, Han W. New development in CAR-T cell therapy.J Hematol Oncol . 2017;10(1):53. doi:10.1186/s13045-017-0423-1
6. Kalos M, June CH. Adoptive T Cell Transfer for Cancer Immunotherapy in the Era of Synthetic Biology. Immunity . 2013;39(1):49-60. doi:10.1016/J.IMMUNI.2013.07.002
7. Sadelain M, Papapetrou EP, Bushman FD. Safe harbours for the integration of new DNA in the human genome. Nat Rev Cancer . 2011;12(1):nrc3179. doi:10.1038/nrc3179
8. Finer M, Dull T, Qin L, Farson D, Roberts M. kat: a high-efficiency retroviral transduction system for primary human T lymphocytes.Blood . 1994;83(1).
9. Naldini L, Blömer U, Gallay P, et al. In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector.Science (80- ) . 272:263-267. doi:10.2307/2889637
10. Scholler J, Brady TL, Binder-Scholl G, et al. Decade-long safety and function of retroviral-modified chimeric antigen receptor T cells.Sci Transl Med . 2012;4(132):132ra53. doi:10.1126/scitranslmed.3003761
11. Mitchell RS, Beitzel BF, Schroder ARW, et al. Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences. Michael Emerman, ed. PLoS Biol . 2004;2(8):e234. doi:10.1371/journal.pbio.0020234
12. Vannucci L, Lai M, Chiuppesi F, Ceccherini-Nelli L, Pistello M. Viral vectors: a look back and ahead on gene transfer technology.New Microbiol . 2013;36(1):1-22.
13. Levine BL, Miskin J, Wonnacott K, Keir C. Global Manufacturing of CAR T Cell Therapy. Mol Ther Methods Clin Dev . 2017;4:92-101. doi:10.1016/j.omtm.2016.12.006
14. Paludan SR, Bowie AG. Cellular & Gene Therapy Guidances - Guidance for Industry: Gene Therapy Clinical Trials - Observing Subjects for Delayed Adverse Events. US Dep Heal Hum Serv Food Drug Adm Cent Biol Eval Res . 2006.
15. Gill S, June CH. Going viral: chimeric antigen receptor T-cell therapy for hematological malignancies. Immunol Rev . 2015;263(1):68-89. doi:10.1111/imr.12243
16. Milone MC, Fish JD, Carpenito C, et al. Chimeric receptors containing CD137 signal transduction domains mediate enhanced survival of T cells and increased antileukemic efficacy in vivo. Mol Ther . 2009;17(8):1453-1464. doi:10.1038/mt.2009.83
17. Kochenderfer JN, Dudley ME, Feldman SA, et al. B-cell depletion and remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cells. Blood . 2012;119(12):2709-2720. doi:10.1182/blood-2011-10-384388
18. Dull T, Zufferey R, Kelly M, et al. A third-generation lentivirus vector with a conditional packaging system. J Virol . 1998;72(11):8463-8471.
19. Frigault MJ, Lee J, Basil MC, et al. Identification of chimeric antigen receptors that mediate constitutive or inducible proliferation of T cells. Cancer Immunol Res . 2015;3(4):356-367. doi:10.1158/2326-6066.CIR-14-0186
20. Zhao N, Qi J, Zeng Z, et al. Transfecting the hard-to-transfect lymphoma/leukemia cells using a simple cationic polymer nanocomplex.J Control Release . 2012;159(1):104-110. doi:10.1016/j.jconrel.2012.01.007
21. Mitchell DA, Karikari I, Cui X, Xie W, Schmittling R, Sampson JH. Selective modification of antigen-specific T cells by RNA electroporation. Hum Gene Ther . 2008;19(5):511-521. doi:10.1089/hum.2007.115
22. Liu L, Johnson C, Fujimura S, Teque F, Levy JA. Transfection optimization for primary human CD8+ cells. J Immunol Methods . 2011;372(1-2):22-29. doi:10.1016/J.JIM.2011.06.026
23. Ramamoorth M, Narvekar A. Non Viral Vectors in Gene Therapy- An Overview. J Clin Diagn Res . 2015;9(1):GE01. doi:10.7860/JCDR/2015/10443.5394
24. Zhang Z, Qiu S, Zhang X, Chen W. Optimized DNA electroporation for primary human T cell engineering. BMC Biotechnol . 2018;18(1):4. doi:10.1186/s12896-018-0419-0
25. Zhang Z, Qiu S, Zhang X, Chen W. Optimized electroporation of Primary T cells. BMC Biotechnol . 2018;18(4):1-9.
26. Jordan ET, Collins M, Terefe J, Ugozzoli L, Rubio T. Optimizing electroporation conditions in primary and other difficult-to-transfect cells. J Biomol Tech . 2008;19(5):328-334.
27. Goffinet C, Keppler OT. Efficient nonviral gene delivery into primary lymphocytes from rats and mice. FASEB J . 2006;20(3):500-502. doi:10.1096/fj.05-4651fje
28. Ross PC, Hui SW. Lipoplex size is a major determinant of in vitro lipofection efficiency. Gene Ther . 1999;6(4):651-659. doi:10.1038/sj.gt.3300863
29. Varkouhi AK, Scholte M, Storm G, Haisma HJ. Endosomal escape pathways for delivery of biologicals. J Control Release . 2011;151(3):220-228. doi:10.1016/j.jconrel.2010.11.004
30. Mašek T, Vopalenský V, Pospíšek M. The Luc2 gene enhances reliability of bicistronic assays. Open Life Sci . 2013;8(5):423-431. doi:10.2478/s11535-013-0151-z
31. Conese M, Biffi A, Dina G, Marziliano N, Villa A. Comparison between Cationic Polymer and Lipid in Plasmidic DNA Delivery to the Cell Nucleus. Open Gene Ther J . 2009;2:21-28.
32. Bragonzi A, Boletta A, Biffi A, et al. Comparison between cationic polymers and lipids in mediating systemic gene delivery to the lungs.Gene Ther . 1999;6(12):1995-2004. doi:10.1038/sj.gt.3301039
33. Ayoubi TA, Van De Ven WJ. Regulation of gene expression by alternative promoters. FASEB J . 1996;10(4):453-460.
34. Qin JY, Zhang L, Clift KL, et al. Systematic comparison of constitutive promoters and the doxycycline-inducible promoter.PLoS One . 2010;5(5):e10611. doi:10.1371/journal.pone.0010611
35. Chung S, Andersson T, Sonntag K-C, Björklund L, Isacson O, Kim K-S. Analysis of different promoter systems for efficient transgene expression in mouse embryonic stem cell lines. Stem Cells . 2002;20(2):139-145. doi:10.1634/stemcells.20-2-139
36. Lacy-Hulbert A, Thomas R, Li X-P, Lilley CE, Coffin RS, Roes J. Interruption of coding sequences by heterologous introns can enhance the functional expression of recombinant genes. Gene Ther . 2001;8(8):649-653. doi:10.1038/sj.gt.3301440
37. Christensen MD, Elmer JJ, Eaton S, Gonzalez-Malerva L, LaBaer J, Rege K. Kinome-level screening identifies inhibition of polo-like kinase-1 (PLK1) as a target for enhancing non-viral transgene expression. J Control Release . 2015;204:20-29. doi:10.1016/j.jconrel.2015.01.036
38. Sutlu T, Nyström S, Gilljam M, Stellan B, Applequist SE, Alici E. Inhibition of Intracellular Antiviral Defense Mechanisms Augments Lentiviral Transduction of Human Natural Killer Cells: Implications for Gene Therapy. Hum Gene Ther . 2012;23(10):1090-1100. doi:10.1089/hum.2012.080
39. Chen Z-Y, Riu E, He C-Y, Xu H, Kay M a. Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Mol Ther . 2008;16(3):548-556. doi:10.1038/sj.mt.6300399
40. Chen Z. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther . 2003;8(3):495-500. doi:10.1016/S1525-0016(03)00168-0
41. Audouy S, Molema G, de Leij L, Hoekstra D. Serum as a modulator of lipoplex-mediated gene transfection: dependence of amphiphile, cell type and complex stability. J Gene Med . 2000;2(6):465-476. doi:10.1002/1521-2254(200011/12)2:6<465::AID-JGM141>3.0.CO;2-Z
42. Yang J-P, Huang L. Overcoming the Inhibitory Effect of Serum on Lipofection by Increasing the Charge Ratio of Cationic Liposome to DNA . Vol 4.; 1997.
43. Joshee N, Bastola DR, Cheng P-W. Transferrin-Facilitated Lipofection Gene Delivery Strategy: Characterization of the Transfection Complexes and Intracellular Trafficking . Vol 13.; 2002. www.liebertpub.com. Accessed August 21, 2019.
44. Yanagihara K, Cheng H, Cheng P-W. Effects of epidermal growth factor, transferrin, and insulin on lipofection efficiency in human lung carcinoma cells. Cancer Gene Ther . 2000;7(1):59-65. doi:10.1038/sj.cgt.7700092
45. Olden BR, Cheng Y, Yu JL, Pun SH. Cationic polymers for non-viral gene delivery to human T cells. J Control Release . 2018;282:140-147. doi:10.1016/j.jconrel.2018.02.043
46. Van Tendeloo VFI, Willems R, Ponsaerts P, et al. High-level transgene expression in primary human T lymphocytes and adult bone marrow CD34+ cells via electroporation-mediated gene delivery.Gene Ther . 2000;7(16):1431-1437. doi:10.1038/sj.gt.3301252
47. Chicaybam L, Sodre AL, Curzio BA, Bonamino MH. An Efficient Low Cost Method for Gene Transfer to T Lymphocytes. Fang D, ed. PLoS One . 2013;8(3):e60298. doi:10.1371/journal.pone.0060298
48. Zhao Y, Zheng Z, Cohen CJ, et al. High-Efficiency Transfection of Primary Human and Mouse T Lymphocytes Using RNA Electroporation.Mol Ther . 2006;13(1):151-159. doi:10.1016/J.YMTHE.2005.07.688
49. Meaking WS, Edgerton J, Wharton CW, Meldrum RA. Electroporation-induced damage in mammalian cell DNA. BBA - Gene Struct Expr . 1995;1264(3):357-362. doi:10.1016/0167-4781(95)00177-8
50. Piñero J, López-Baena M, Ortiz T, Cortés F. Apoptotic and Necrotic Cell Death Are Both Induced by Electroporation in HL60 Human Promyeloid Leukaemia Cells . Vol 2.; 1997.
51. Ortiz T, Piñero J, Cortés F. Chromosome damage induced by combined treatments with restriction endonucleases introduced into CHO cells by single or double electroporation. Mutat Res - Fundam Mol Mech Mutagen . 1995;327(1-2):161-169. doi:10.1016/0027-5107(94)00183-6
52. Lesueur L, Mir LM, Andr FM. Overcoming the Specific Toxicity of Large Plasmids Electrotransfer in Primary Cells In Vitro. 2016. doi:10.1038/mtna.2016.4
53. Pradhan K, Gadgil M. Effect of addition of “carrier” DNA during transient protein expression in suspension CHO culture.Cytotechnology . 2012;64(6):613-622. doi:10.1007/s10616-012-9435-4
54. Sakurai H, Kawabata K, Sakurai F, Nakagawa S, Mizuguchi H. Innate immune response induced by gene delivery vectors. Int J Pharm . 2008;354(1-2):9-15. doi:10.1016/j.ijpharm.2007.06.012
55. Chakrabarti A, Jha BK, Silverman RH. New insights into the role of RNase L in innate immunity. J Interferon Cytokine Res . 2011;31(1):49-57. doi:10.1089/jir.2010.0120
56. Atianand MK, Fitzgerald KA. Molecular basis of DNA recognition in the immune system. J Immunol . 2013;190(5):1911-1918. doi:10.4049/jimmunol.1203162
57. Christianson HC, Belting M. Heparan sulfate proteoglycan as a cell-surface endocytosis receptor. Matrix Biol . 2014;35:51-55. doi:10.1016/j.matbio.2013.10.004
58. Teixé T, Nieto-Blanco P, Vilella R, Engel P, Reina M, Espel E. Syndecan-2 and -4 expressed on activated primary human CD4+ lymphocytes can regulate T cell activation. Mol Immunol . 2008;45(10):2905-2919. doi:10.1016/j.molimm.2008.01.033
59. Fadnes B, Husebekk A, Svineng G, et al. The proteoglycan repertoire of lymphoid cells. Glycoconj J . 2012;29(7):513-523. doi:10.1007/s10719-012-9427-9
60. Subramanian Vignesh K, Deepe GS, Jr. Metallothioneins: Emerging Modulators in Immunity and Infection. Int J Mol Sci . 2017;18(10). doi:10.3390/ijms18102197
61. Rice JM, Zweifach A, Lynes MA. Metallothionein regulates intracellular zinc signaling during CD4 + T cell activation. 2016. doi:10.1186/s12865-016-0151-2
62. Wu C, Pot C, Apetoh L, et al. Metallothioneins negatively regulate IL-27-induced type 1 regulatory T-cell differentiation. Proc Natl Acad Sci U S A . 2013;110(19):7802-7807. doi:10.1073/pnas.1211776110
63. Doitsh G, Galloway NLK, Geng X, et al. Cell death by pyroptosis drives CD4 T-cell depletion in HIV-1 infection. Nature . 2014;505(7484):509-514. doi:10.1038/nature12940
64. Muñoz-Arias I, Doitsh G, Yang Z, Sowinski S, Ruelas D, Greene WC. Blood-Derived CD4 T Cells Naturally Resist Pyroptosis during Abortive HIV-1 Infection. Cell Host Microbe . 2015;18(4):463-470. doi:10.1016/j.chom.2015.09.010
65. Ng YC, Chung W-C, Kang H-R, et al. A DNA-sensing-independent role of a nuclear RNA helicase, DHX9, in stimulation of NF-B-mediated innate immunity against DNA virus infection. Nucleic Acids Res . 2018;46(17):9011-9026. doi:10.1093/nar/gky742
66. Zhang X, Brann TW, Zhou M, et al. Cutting Edge: Ku70 Is a Novel Cytosolic DNA Sensor That Induces Type III Rather Than Type I IFN.J Immunol . 2011;186(8):4541-4545. doi:10.4049/jimmunol.1003389
67. Letoha T, Keller-Pintér A, Kusz E, et al. Cell-penetrating peptide exploited syndecans. Biochim Biophys Acta - Biomembr . 2010;1798(12):2258-2265. doi:10.1016/j.bbamem.2010.01.022
68. Qin L, Ding Y, Pahud DR, Chang E, Imperiale MJ, Bromberg JS. Promoter attenuation in gene therapy: Interferon-γ and tumor necrosis factor-α inhibit transgene expression. Hum Gene Ther . 1997;8(17):2019-2029. doi:10.1089/hum.1997.8.17-2019
69. Pine R. Constitutive expression of an ISGF2/IRF1 transgene leads to interferon-independent activation of interferon-inducible genes and resistance to virus infection. J Virol . 1992;66(7):4470-4478. doi:10.1128/jvi.66.7.4470-4478.1992
70. Sellins K, Fradkin L, Liggitt D, Dow S. Type I interferons potently suppress gene expression following gene delivery using liposome-DNA complexes. Mol Ther . 2005;12(3):451-459. doi:10.1016/j.ymthe.2005.04.008
71. Nakazawa Y, Huye LE, Dotti G, et al. Optimization of the piggybac transposon system for the sustained genetic modification of human T lymphocytes. J Immunother . 2009;32(8):826-836. doi:10.1097/CJI.0b013e3181ad762b
72. Roth TL, Puig-Saus C, Yu R, et al. Reprogramming human T cell function and specificity with non-viral genome targeting. Nature . 2018;559(7714):405-409. doi:10.1038/s41586-018-0326-5
73. Olden BR, Cheng E, Cheng Y, Pun SH. Identifying key barriers in cationic polymer gene delivery to human T cells. Biomater Sci . 2019;7(3):789-797. doi:10.1039/c8bm01262h
74. Pampusch MS, Haran KP, Hart GT, et al. Rapid Transduction and Expansion of Transduced T Cells with Maintenance of Central Memory Populations. Mol Ther - Methods Clin Dev . 2020;16:1-10. doi:10.1016/j.omtm.2019.09.007
75. Tumeh PC, Koya RC, Chodon T, et al. The impact of ex vivo clinical grade activation protocols on human T cell phenotype and function for the generation of genetically modified cells for adoptive cell transfer therapy. J Immunother . 2010;33(8):759-768. doi:10.1097/CJI.0b013e3181f1d644
76. Bai H, Lester GMS, Petishnok LC, Dean DA. Cytoplasmic transport and nuclear import of plasmid DNA. Biosci Rep . 2017;37(6). doi:10.1042/BSR20160616
77. Bobardt MD, Armand-Ugón M, Clotet I, et al. Effect of polyanion-resistance on HIV-1 infection. Virology . 2004;325(2):389-398. doi:10.1016/j.virol.2004.05.011
78. Sarrazin S, Lamanna WC, Esko JD. Heparan sulfate proteoglycans.Cold Spring Harb Perspect Biol . 2011;3(7):1-33. doi:10.1101/cshperspect.a004952
79. Spillmann D. Heparan sulfate: anchor for viral intruders?Biochimie . 2001;83(8):811-817. doi:10.1016/s0300-9084(01)01290-1
80. Bastien Paris S, Burlacu A, Durocher Y. Opposing Roles of Syndecan-1 and Syndecan-2 in Polyethyleneimine-mediated Gene Delivery * □ S. 2008. doi:10.1074/jbc.M705424200
81. Maude SL, Teachey DT, Rheingold SR, et al. Sustained remissions with CD19-specific chimeric antigen receptor (CAR)-modified T cells in children with relapsed/refractory ALL. J Clin Oncol. 2016;34(15_suppl):3011-3011. doi:10.1200/jco.2016.34.15_suppl.3011
82. Moffett, H. F., Coon, M. E., Radtke, S., Stephan, S. B., McKnight, L., Lambert, A., Stoddard, B. L., Kiem, H. P. & Stephan, M. T. Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers. Nat. Commun. 8, 1–13 (2017).
83. Smith, T. T., Stephan, S. B., Moffett, H. F., McKnight, L. E., Ji, W., Reiman, D., Bonagofski, E., Wohlfahrt, M. E., Pillai, S. P. S. & Stephan, M. T. In situ programming of leukaemia-specific t cells using synthetic DNA nanocarriers. Nat. Nanotechnol. 12, 813–822 (2017).
84. Raup, A., Stahlschmidt, U., Jérôme, V., Synatschke, C., Müller, A. & Freitag, R. Influence of Polyplex Formation on the Performance of Star-Shaped Polycationic Transfection Agents for Mammalian Cells. Polymers (Basel). 8, 224 (2016).
85. Schallon, A., Synatschke, C. V., Jérôme, V., Müller, A. H. E. & Freitag, R. Nanoparticulate nonviral agent for the effective delivery of pDNA and siRNA to differentiated cells and primary human T lymphocytes. Biomacromolecules 13, 3463–3474 (2012).
85. Xie, Y., Kim, N. H., Nadithe, V., Schalk, D., Thakur, A., Kılıç, A., Lum, L. G., Bassett, D. J. P. & Merkel, O. M. Targeted delivery of siRNA to activated T cells via transferrin-polyethylenimine (Tf-PEI) as a potential therapy of asthma. J. Control. Release 229, 120–129 (2016).
87. Olden, B. R., Cheng, Y., Yu, J. L. & Pun, S. H. Cationic polymers for non-viral gene delivery to human T cells. J. Control. Release 282, 140–147 (2018).