T-cell gene therapy

Current Opinion in Biotechnology

Volumn 7, Issue 6, 1996, Pages 629-634

  Hege, Kristen M ^a   Roberts, Margo R ^a  

^a CELL GENESYS INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; T LYMPHOCYTE RECEPTOR;

ADOPTIVE IMMUNOTHERAPY; ADOPTIVE TRANSFER; ANTIGEN SPECIFICITY; CANCER IMMUNOTHERAPY; CLINICAL TRIAL; COMBINED IMMUNODEFICIENCY; CYTOTOXIC T LYMPHOCYTE; ENZYME DEFICIENCY; GENE THERAPY; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; PRIORITY JOURNAL; SHORT SURVEY; T LYMPHOCYTE; VIRUS INFECTION; VIRUS RESISTANCE;

HUMAN HERPESVIRUS 4; HUMAN IMMUNODEFICIENCY VIRUS;

EID: 0030561024     PISSN: 09581669     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0958-1669(96)80074-7     Document Type: Article

References (53)

1. Walter EA, Greenberg PD, Gilbert MJ, Finch RJ, Watanabe MS, Thomas ED, Riddell SR. Reconstitution of cellular immunity against cytomegalovirus in recipients of allogeneic bone marrow by transfer of T-cell clones from the donor. of outstanding interest N Engl J Med. 333:1995;1038-1044 This report is a follow up of a 1992 report and confirms the reconstitution of cellular immunity to CMV by adoptive transfer of CMV-specific CTLs in 14 patients undergoing bone marrow transplantation. Clones persisted in vivo for at least 12 weeks but CTL activity declined in patients deficient in a CMV-specific CD4 immune response.
2. Riddell S, Watanabe KS, Goodrich JM, Li CR, Agha ME, Greenberg PD. Restoration of viral immunity in immunodeficient humans by the adoptive transfer of T cell clones. Science. 257:1992;238-241.
3. Rosenberg SA, Packard BS, Aebersold PM, Soloman D, Topalian SL, Toy ST, Simon P, Lotze MT, Yang JC, Seipp CA. Use of tumor-infiltrating lymphocytes and interleukin-2 in the immunotherapy of patients with metastatic melanoma. A preliminary report. N Engl J Med. 319:1988;1676-1680.
4. Baltimore D. Intracellular immunization. Nature. 335:1988;395-396.
5. Friedman AD, Triezenberg SJ, McKnight SL. Expression of a truncated viral transactivator selectively impedes lytic infection by its cognate virus. Nature. 335:1988;452-454.
6. + T-cell deficient mice. J Virol. 68:1994;4700-4704.
7. c chain sufficient for Jak kinase activation and induction of proliferation in T cells. Mol Cell Biol. 16:1996;309-317.
8. Nelson BH, Lord JD, Greenberg PD. Cytoplasmic domains of the interleukin-2 receptor β and γ chains mediate the signal for T cell proliferation. Nature. 369:1994;333-336.
9. Riddell SR, Greenberg PD. Principles for adoptive T cell therapy of human viral diseases. Annu Rev Immunol. 13:1995;545-586.
10. Papadopoulos EB, Ladanyi M, Emanuel D, Mackinnon S, Boulard F, Carabasi MH, Castro-Malaspina H, Childs BH, Gillio AP, Small TN, et al. Infusions of donor leukocytes to treat Epstein - Barr virus-associated lymphoproliferative disorders after allogeneic bone marrow transplantation. N Engl J Med. 330:1994;1185-1191.
11. r-marked EBV-specific T cells into immuno-deficient bone marrow transplant recipients established populations of CTL precursors that responded to in vivo or ex vivo challenge with EBV for up to 18 months.
12. r-marked EBV-specific CTL inhibited EBV reactivation and promoted regression of an established EBV-associated lymphoma in patients undergoing bone marrow tranplantation. Adoptively transferred T cells persisted for at least 10 weeks.
13. + T-cell clone. HIV suppressive activity of this supernatant was completely blocked by antibodies to all three chemokines. Recombinant chemokines inhibited HIV and simian immunodeficiency virus infection.
14. Riddell SR, Elliot M, Lewinsohn DA, Gilbert MJ, Wilson L, Manley SA, Lupton SD, Overell RW, Reynolds TC, Corey L, et al. T cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. of outstanding interest Nat Med. 2:1996;216-223 The infusion of autologous gag-specific CTLs expressing a chimeric suicide gene into HIV-infected patients resulted in the development of a cellular immune response to the novel protein and elimination of the transduced T cells in five out of six patients. The suicide gene was constructed from hygromycin phosphotransferase and herpes virus thymidine kinase to allow for both positive and negative selection of transduced cells.
15. + HIV-specific T cells for HIV seropositive patients undergoing allogeneic bone marrow transplant. Hum Gene Ther. 3:1992;319-338.
16. Fruh K. A viral inhibitor of peptide transporters for antigen presentation. Nature. 375:1995;415-418.
17. Hill A. Herpes simplex virus turns off the TAP to evade host immunity. Nature. 375:1995;411-415.
18. Koenig S, Conley AJ, Brewah YA, Jones GM, Leath S, Boots LJ, Davey V, Pantaleo G, Demarest JF, Carter C. Transfer of HIV-1 specific cytotoxic T lymphocytes to an AIDS patient leads to selection for mutant HIV variants and subsequent disease progression. of special interest Nat Med. 1:1995;330-336 This is a disturbing report of the disease progression triggered by the infusion of autologous HIV-1 nef-specific CTLs into an HIV-infected patient. Nef-deleted viral mutants were recovered from the patient post infusion.
19. + T lymphocytes armed with Universal T-cell receptors. Blood. 84:1994;2878-2889.
20. Tran A-C, Zhang D, Byrn R, Roberts MR. Chimeric ζ-receptors direct human natural killer effector function to permit killing of NK-resistant tumor cells and HIV-infected T lymphocytes. J Immunol. 155:1994;1000-1009.
21. Finer MH, Dull TJ, Qin L, Farson D, Roberts MR. kat: a high-efficiency retroviral transduction system for primary human T lymphocytes. Blood. 83:1994;43-50.
22. Walker R, Bechtel CM, Natarajan V, Baseler M, Metcalf JA, Stevens R, Blaese RM, Davey RT, Falloon J, Polis MA, et al. Adoptive transfer of genetically modified, HIV-1 specific, syngeneic cytotoxic T lymphocytes (CTL) in HIV discordant identical twins. Abstract 404 of the 3rd Conference on Retroviruses and Opportunistic Infections. 1995 January 28-February 1;. Washington, DC.
23. Lisziewicz J, Sun D, Lisziewicz A, Gallo RC. Antitat gene therapy: a candidate for late-stage AIDS patients. Gene Ther. 2:1995;218-222.
24. Escaich S, Kalfoglou C, Plavec I, Kaushal S, Mosca J, Bohnlein E. RevM10-mediated inhibition of HIV-1 replication in chronically infected T cells. Hum Gene Ther. 6:1995;625-634.
25. Woffendin C, Yang Z-Y, Udaykumar R, Xu L, Yang N-S, Sheehy MJ, Nabel GJ. Nonviral and viral delivery of a human immunodeficiency virus protective gene into primary human T cells. Proc Natl Acad Sci USA. 91:1994;11581-11585.
26. + cord blood progenitors were transduced with an HIV-1 specific ribozyme. Gene-modified cells were resistant to infection with a macrophage-tropic strain of HIV.
27. Yamada O, Yu M, Yee J-K, Kraus G, Looney D, Wong-Staal F. Intracellular immunization of human T cells with a hairpin ribozyme against human immunodeficiency virus type 1. Gene Ther. 1:1994;38-45.
28. Leavitt MC, Yu M, Yamada O, Kraus G, Looney D, Poeschla E, Wong-Staal F. Transfer of an anti-HIV-1 ribozyme gene into primary human lymphocytes. Hum Gene Ther. 5:1994;1115-1120.
29. Zhou C, Bahner IC, Larson GP, Zaia JA, Rossi JJ, Kohn DB. Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-tat and rev hammerhead ribozymes. Gene. 149:1994;33-39.
30. + T lymphocytes. Hum Gene Ther. 5:1994;193-201.
31. Lee S-W, Gallardo HF, Gilboa E, Smith C. Inhibition of human immunodeficiency virus type-1 in human T cells by a potent rev response element decoy consisting of the 13-nucleotide minimal rev-binding domain. J Virol. 68:1994;8254-8264.
32. Maciejewski JP, Weichold FF, Young NS, Cara A, Zella D, Reitz MS, Gallo RC. Intracellular expression of antibody fragments directed against HIV reverse transcriptase prevents HIV infection in vitro. of special interest Nat Med. 1:1995;667-673 A lymphoblastoid cell line transduced with an anti-reverse transcriptase Fab was resistant to infection with various isolates of HIV-1 and HIV-2 at a multiplicity of infection (m.o.i.) of 2.0.
33. Duan L, Bagasra O, Laughlin MA, Oakes JW, Pomerantz RI. Potent inhibition of human immunodeficiency virus type 1 replication by an intracellular anti-Rev single chain antibody. Proc Natl Acad Sci USA. 91:1994;5015-5019.
34. Woffendin C, Udaykumar R, Yang Z-Y, Xu L, Nabel GJ. Expression of a protective gene prolongs survival of T cells in human immunodeficiency virus-infected patients. of outstanding interest Proc Natl Acad Sci USA. 93:1996;2889-2894 This was the first human trial of adoptively transferred T cells genetically modified to express an HIV resistance gene. T cells expressing Rev M10 had modestly improved in vivo survival compared to cells transduced with a control vector in two patients.
35. Kolb HJ, Schattenberg A, Goldman JM, Ferrant A, Verdonck L, Niederwieser D, et al. Graft-versus-leukemia effect of donor lymphocyte transfusions in marrow grafted patients. of special interest Blood. 86:1995;2041-2050 Donor lymphocyte infusions in 135 patients with relapsed malignancy following allogeneic bone marrow transplant are described. Complete remissions were induced in chronic myeloid leukemia (73%), acute myeloid leukemia (29%), and one patient each with polycythemia vera and myelodysplasia.
36. Rosenberg WA. The immunotherapy and gene therapy of cancer. J Clin Oncol. 10:1992;180-199.
37. Rosenberg SA, Aebersold P, Cornetta K. Gene transfer into humans - immunotherapy of patients with advanced melanoma using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med. 323:1990;570-578.
38. Griffith KD, Read EJ, Carrasquillo JA, Carter CS, Yang JC, Fisher B, Aebersold P, Packard BS, Yu MY, Rosenberg SA. In vivo distribution of adoptively transferred indium-111 labeled tumor infiltrating lymphocytes in patients with metastatic melanoma. J Natl Cancer Inst. 81:1989;1709-1717.
39. Fisher B, Packard B, Read E. Tumor localization of adoptively transferred indium-111 labeled tumor infiltrating lymphocytes in patients with metastatic melanoma. J Clin Oncol. 7:1989;250-261.
40. Treisman J, Hwu P, Yannelli JR, Shafer GE, Cowherd R, Samid D, Rosenberg S. Upregulation of tumor necrosis factor-α production by retrovirally transduced human tumor-infiltrating lymphocytes using trans-retinoic acid. Cell Immunol. 156:1994;448-457.
41. Hwu P, Yannelli J, Kriegler M. Functional and molecular characterization of tumor-infiltrating lymphocytes transduced with tumor necrosis factor-α cDNA for the gene therapy of cancer in humans. J Immunol. 150:1993;4104-4115.
42. Rosenberg SA, Kasid A, Anderson WF. TNF/TIL human gene therapy clinical protocol. Hum Gene Ther. 1:1991;443-462.
43. Hwu P, Rosenberg SA. The genetic modification of T cells for cancer therapy: an overview of laboratory and clinical trials. Cancer Detect Prev. 18:1994;43-50.
44. Nakamura Y, Wakimoto H, Abe J, Kanegae Y, Saito I, Aoyagi M, Hirakawa K, Hamada H. Adoptive immunotherapy with murine tumor-specific T lymphocytes engineered to secrete interleukin 2. Cancer Res. 54:1994;5757-5760.
45. Minamoto S, Treisman J, Hankins WD, Sugamura K, Rosenberg SA. Acquired erythropoietin responsiveness of interleukin-2 dependent T lymphocytes retrovirally transduced with genes encoding chimeric erythropoietin/interleukin-2 receptors. of special interest Blood. 86:1995;2281-2287 The transduction of an IL-2-dependent T-cell line with chimeric receptors composed of the extracellular domain of the EpoR and the IL-2R β or γ chains (EβR and EγR) stimulated Epo-dependent proliferation. Infection with both EβR and EγR was required to induce Epo dependency.
46. Fujita K, Ikarashi H, Takakuwa K, Kodama S, Akai T, Yamamoto T, Tanaka K. Enhancement of T cell receptor signaling of tumor-infiltrating lymphocytes of retrovirally mediated fyn gene transduction. Jpn J Cancer Res. 85:1994;1073-1079.
47. Cole DJ, Weil DP, Shilyanska J, Custer M, Kawakami Y, Rosenberg SA, Nishimura MI. Characterization of the functional specificity of a cloned T cell receptor heterodimer recognizing the MART-1 melanoma antigen. Cancer Res. 55:1995;748-752.
48. Hwu P, Shafer GE, Treisman J, Schindler DG, Gross G, Cowherd R, Rosenberg SA, Eshhar Z. Lysis of ovarian cancer cells by human lymphocytes redirected with a chimeric gene composed of an antibody variable region and the Fc receptor γ chain. J Exp Med. 178:1993;361-366.
49. Eshhar Z, Waks T, Gross G, Schindler D. Specific activation and targeting of cytotoxic lymphocytes through chimeric single chains consisting of antibody-binding domains and the γ or ζ subunits of the immunoglobulin and T-cell receptors. Proc Natl Acad Sci USA. 90:1993;720-724.
50. Hwu P, Yang C, Cowherd R, Treisman J, Shafer GE, Eshhar Z, Rosenberg SA. In vivo antitumor activity of T cells redirected with chimeric antibody/T-cell receptor genes. of special interest Cancer Res. 55:1995;3369-3373 Murine TIL transduced with MOv-γ, a chimeric receptor directed against ovarian carcinoma cells, demonstrated efficacy in two mouse tumor models in which modified T cells and tumor targets were injected via either the intraperitoneal or the intravenous route.
51. Moritz D, Groner B. A spacer region between the single chain antibody- and the CD3 zeta-chain domain of chimeric T cell receptor components is required for efficient ligand binding and signaling activity. Gene Ther. 2:1995;539-546.
52. Blaese RM, Culver KW, Miller D, 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. of outstanding interest Science. 270:1995;475-480 The four-year follow up of ADA-gene transfer into the peripheral blood lymphocytes of two children with SCID is reported. An improvement in immune function was seen in both despite the lack of the improvement in measurable ADA enzyme levels in one.
53. Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P, Mazzolari E, Maggioni D, Rossi C, Servida P, et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. of outstanding interest Science. 270:1995;470-475 The clinical trial results of ADA gene transfer into the peripheral blood lymphocytes and bone marrow of two infants with SCID are reported. The long-term survival of ADA-expressing cells was demonstrated along with an improvement in cellular and humoral immunity in both patients. The long-term reconstitution of ADA-modified cells resulted almost exclusively from transduced bone marrow derived progenitor cells.