Gene-modified hematopoietic stem cells for cancer immunotherapy

References

• Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995; 270:475 - 80; http://dx.doi.org/10.1126/science.270.5235.475; PMID: 7570001
   PubMed Web of Science ®Google Scholar
• Brentjens RJ, Rivière I, Park JH, Davila ML, Wang X, Stefanski J, Taylor C, Yeh R, Bartido S, Borquez-Ojeda O, et al. Safety and persistence of adoptively transferred autologous CD19-targeted T cells in patients with relapsed or chemotherapy refractory B-cell leukemias. Blood 2011; 118:4817 - 28; http://dx.doi.org/10.1182/blood-2011-04-348540; PMID: 21849486
   PubMed Web of Science ®Google Scholar
• Kochenderfer JN, Dudley ME, Feldman SA, Wilson WH, Spaner DE, Maric I, Stetler-Stevenson M, Phan GQ, Hughes MS, Sherry RM, 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; 119:2709 - 20; http://dx.doi.org/10.1182/blood-2011-10-384388; PMID: 22160384
   PubMed Web of Science ®Google Scholar
• Grupp SA, Kalos M, Barrett D, Aplenc R, Porter DL, Rheingold SR, Teachey DT, Chew A, Hauck B, Wright JF, et al. Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. N Engl J Med 2013; 368:1509 - 18; http://dx.doi.org/10.1056/NEJMoa1215134; PMID: 23527958
   PubMed Web of Science ®Google Scholar
• Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009; 326:818 - 23; http://dx.doi.org/10.1126/science.1171242; PMID: 19892975
   PubMed Web of Science ®Google Scholar
• Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2010; 363:355 - 64; http://dx.doi.org/10.1056/NEJMoa1000164; PMID: 20660403
   PubMed Web of Science ®Google Scholar
• Kohn DB, Pai SY, Sadelain M. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells. Biol Blood Marrow Transplant 2013; 19:Suppl S64 - 9; http://dx.doi.org/10.1016/j.bbmt.2012.09.021; PMID: 23032601
   PubMedGoogle Scholar
• Boztug K, Schmidt M, Schwarzer A, Banerjee PP, Díez IA, Dewey RA, Böhm M, Nowrouzi A, Ball CR, Glimm H, et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 2010; 363:1918 - 27; http://dx.doi.org/10.1056/NEJMoa1003548; PMID: 21067383
   PubMed Web of Science ®Google Scholar
• Rivat C, Santilli G, Gaspar HB, Thrasher AJ. Gene therapy for primary immunodeficiencies. Hum Gene Ther 2012; 23:668 - 75; http://dx.doi.org/10.1089/hum.2012.116; PMID: 22691036
   PubMed Web of Science ®Google Scholar
• Robbins PF, Morgan RA, Feldman SA, Yang JC, Sherry RM, Dudley ME, Wunderlich JR, Nahvi AV, Helman LJ, Mackall CL, et al. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol 2011; 29:917 - 24; http://dx.doi.org/10.1200/JCO.2010.32.2537; PMID: 21282551
   PubMed Web of Science ®Google Scholar
• van Lent AU, Nagasawa M, van Loenen MM, Schotte R, Schumacher TN, Heemskerk MH, Spits H, Legrand N. Functional human antigen-specific T cells produced in vitro using retroviral T cell receptor transfer into hematopoietic progenitors. J Immunol 2007; 179:4959 - 68; PMID: 17911580
   PubMed Web of Science ®Google Scholar
• Zhao Y, Parkhurst MR, Zheng Z, Cohen CJ, Riley JP, Gattinoni L, Restifo NP, Rosenberg SA, Morgan RA. Extrathymic generation of tumor-specific T cells from genetically engineered human hematopoietic stem cells via Notch signaling. Cancer Res 2007; 67:2425 - 9; http://dx.doi.org/10.1158/0008-5472.CAN-06-3977; PMID: 17363559
   PubMed Web of Science ®Google Scholar
• Vatakis DN, Koya RC, Nixon CC, Wei L, Kim SG, Avancena P, Bristol G, Baltimore D, Kohn DB, Ribas A, et al. Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proc Natl Acad Sci U S A 2011; 108:E1408 - 16; http://dx.doi.org/10.1073/pnas.1115050108; PMID: 22123951
   PubMedGoogle Scholar
• Bendle GM, Linnemann C, Hooijkaas AI, Bies L, de Witte MA, Jorritsma A, Kaiser AD, Pouw N, Debets R, Kieback E, et al. Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy. Nat Med 2010; 16:565 - 70, 1p, 570; http://dx.doi.org/10.1038/nm.2128; PMID: 20400962
   PubMed Web of Science ®Google Scholar
• Giannoni F, Hardee CL, Wherley J, Gschweng E, Senadheera S, Kaufman ML, Chan R, Bahner I, Gersuk V, Wang X, et al. Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells. Mol Ther 2013; 21:1044 - 54; http://dx.doi.org/10.1038/mt.2013.8; PMID: 23380815
   PubMed Web of Science ®Google Scholar
• Roberts MR, Cooke KS, Tran AC, Smith KA, Lin WY, Wang M, Dull TJ, Farson D, Zsebo KM, Finer MH. Antigen-specific cytolysis by neutrophils and NK cells expressing chimeric immune receptors bearing zeta or gamma signaling domains. J Immunol 1998; 161:375 - 84; PMID: 9647246
   PubMed Web of Science ®Google Scholar
• Badowski MS, Zhang T, Tsang TC, Harris DT. Chimeric antigen receptors for stem cell based immunotherapy. J Exp Ther Oncol 2009; 8:53 - 63; PMID: 19827271
   PubMedGoogle Scholar
• Li L, Liu LN, Feller S, Allen C, Shivakumar R, Fratantoni J, Wolfraim LA, Fujisaki H, Campana D, Chopas N, et al. Expression of chimeric antigen receptors in natural killer cells with a regulatory-compliant non-viral method. Cancer Gene Ther 2010; 17:147 - 54; http://dx.doi.org/10.1038/cgt.2009.61; PMID: 19745843
   PubMed Web of Science ®Google Scholar
• Schirrmann T, Pecher G. Specific targeting of CD33(+) leukemia cells by a natural killer cell line modified with a chimeric receptor. Leuk Res 2005; 29:301 - 6; http://dx.doi.org/10.1016/j.leukres.2004.07.005; PMID: 15661266
   PubMed Web of Science ®Google Scholar
• Kowolik CM, Topp MS, Gonzalez S, Pfeiffer T, Olivares S, Gonzalez N, Smith DD, Forman SJ, Jensen MC, Cooper LJ. CD28 costimulation provided through a CD19-specific chimeric antigen receptor enhances in vivo persistence and antitumor efficacy of adoptively transferred T cells. Cancer Res 2006; 66:10995 - 1004; http://dx.doi.org/10.1158/0008-5472.CAN-06-0160; PMID: 17108138
   PubMed Web of Science ®Google Scholar
• Savoldo B, Ramos CA, Liu E, Mims MP, Keating MJ, Carrum G, Kamble RT, Bollard CM, Gee AP, Mei Z, et al. CD28 costimulation improves expansion and persistence of chimeric antigen receptor-modified T cells in lymphoma patients. J Clin Invest 2011; 121:1822 - 6; http://dx.doi.org/10.1172/JCI46110; PMID: 21540550
   PubMed Web of Science ®Google Scholar
• Brentjens RJ, Davila ML, Riviere I, Park J, Wang X, Cowell LG, Bartido S, Stefanski J, Taylor C, Olszewska M, et al. CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia. Sci Transl Med 2013; 5:77ra38; http://dx.doi.org/10.1126/scitranslmed.3005930; PMID: 23515080
   PubMed Web of Science ®Google Scholar
• Kochenderfer JN, Dudley ME, Carpenter RO, Kassim SH, Rose JJ, Telford WG, Hakim FT, Halverson DC, Fowler DH, Hardy NM, et al. Donor-derived CD19-targeted T cells cause regression of malignancy persisting after allogeneic hematopoietic stem cell transplantation. [Epub ahead of print] Blood 2013; 122:4129 - 39; http://dx.doi.org/10.1182/blood-2013-08-519413; PMID: 24055823
   PubMed Web of Science ®Google Scholar
• De Oliveira SN, Ryan C, Giannoni F, Hardee CL, Tremcinska I, Katebian B, Wherley J, Sahaghian A, Tu A, Grogan T, et al. Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Hum Gene Ther 2013; 24:824 - 39; http://dx.doi.org/10.1089/hum.2012.202; PMID: 23978226
   PubMed Web of Science ®Google Scholar
• Larson SM, Tu A, Senadheera S, et al. Anti-CD19 chimeric antigen receptor controlled by the suicide gene HSVsr39TK in hematopoietic stem cells for immunotherapy of B-lineage malignancies. American Society of Hematology Annual Meeting. 2013. Abstract 1659.
   Google Scholar
• Bonini C, Ferrari G, Verzeletti S, Servida P, Zappone E, Ruggieri L, Ponzoni M, Rossini S, Mavilio F, Traversari C, et al. HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 1997; 276:1719 - 24; http://dx.doi.org/10.1126/science.276.5319.1719; PMID: 9180086
   PubMed Web of Science ®Google Scholar
• Straathof KC, Pulè MA, Yotnda P, Dotti G, Vanin EF, Brenner MK, Heslop HE, Spencer DM, Rooney CM. An inducible caspase 9 safety switch for T-cell therapy. Blood 2005; 105:4247 - 54; http://dx.doi.org/10.1182/blood-2004-11-4564; PMID: 15728125
   PubMed Web of Science ®Google Scholar
• Serafini M, Manganini M, Borleri G, Bonamino M, Imberti L, Biondi A, Golay J, Rambaldi A, Introna M. Characterization of CD20-transduced T lymphocytes as an alternative suicide gene therapy approach for the treatment of graft-versus-host disease. Hum Gene Ther 2004; 15:63 - 76; http://dx.doi.org/10.1089/10430340460732463; PMID: 14965378
   PubMed Web of Science ®Google Scholar
• Wang X, Chang WC, Wong CW, Colcher D, Sherman M, Ostberg JR, Forman SJ, Riddell SR, Jensen MC. A transgene-encoded cell surface polypeptide for selection, in vivo tracking, and ablation of engineered cells. Blood 2011; 118:1255 - 63; http://dx.doi.org/10.1182/blood-2011-02-337360; PMID: 21653320
   PubMed Web of Science ®Google Scholar
• Traversari C, Marktel S, Magnani Z, Mangia P, Russo V, Ciceri F, Bonini C, Bordignon C. The potential immunogenicity of the TK suicide gene does not prevent full clinical benefit associated with the use of TK-transduced donor lymphocytes in HSCT for hematologic malignancies. Blood 2007; 109:4708 - 15; http://dx.doi.org/10.1182/blood-2006-04-015230; PMID: 17327417
   PubMed Web of Science ®Google Scholar
• Ciceri F, Bonini C, Stanghellini MT, Bondanza A, Traversari C, Salomoni M, Turchetto L, Colombi S, Bernardi M, Peccatori J, et al. Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study. Lancet Oncol 2009; 10:489 - 500; http://dx.doi.org/10.1016/S1470-2045(09)70074-9; PMID: 19345145
   PubMed Web of Science ®Google Scholar
• Barese CN, Krouse AE, Metzger ME, King CA, Traversari C, Marini FC, Donahue RE, Dunbar CE. Thymidine kinase suicide gene-mediated ganciclovir ablation of autologous gene-modified rhesus hematopoiesis. Mol Ther 2012; 20:1932 - 43; http://dx.doi.org/10.1038/mt.2012.166; PMID: 22910293
   PubMed Web of Science ®Google Scholar
• Di Stasi A, Tey SK, Dotti G, Fujita Y, Kennedy-Nasser A, Martinez C, Straathof K, Liu E, Durett AG, Grilley B, et al. Inducible apoptosis as a safety switch for adoptive cell therapy. N Engl J Med 2011; 365:1673 - 83; http://dx.doi.org/10.1056/NEJMoa1106152; PMID: 22047558
   PubMed Web of Science ®Google Scholar