Diphtheria toxin a gene-mediated HIV-1 protection of cord blood-derived T cells in the SCID-hu mouse model

Journal of Hematotherapy and Stem Cell Research

Volumn 7, Issue 4, 1998, Pages 319-331

  Banda, Nirmal K ^a   Akkina, Ramesh K ^b   Terrell, Kristina ^a   Shpall, Elizabeth J ^a   Tomczak, Jennifer ^a   Campain, Julie ^a   Claman, Henry ^a   Cagle, Linda ^a   Harrison, Gail Singer ^a,c  

^a UNIVERSITY OF COLORADO   (United States)

^b Immunology and Pathology   (United States)

^c UNIVERSITY OF COLORADO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DIPHTHERIA TOXIN;

ANIMAL MODEL; ARTICLE; COMBINED IMMUNODEFICIENCY; GENE THERAPY; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; MOUSE; NONHUMAN; PRIORITY JOURNAL; TREATMENT INDICATION; UMBILICAL CORD BLOOD;

ANIMALS; CYTOTOXICITY, IMMUNOLOGIC; DIPHTHERIA TOXIN; FETAL BLOOD; FETAL TISSUE TRANSPLANTATION; GENE TRANSFER TECHNIQUES; HIV INFECTIONS; HIV-1; HUMANS; MICE; MICE, SCID; PEPTIDE FRAGMENTS; T-LYMPHOCYTES;

EID: 0031681424     PISSN: 15258165     EISSN: None     Source Type: Journal    
DOI: 10.1089/scd.1.1998.7.319     Document Type: Article

References (67)

1. Pantaleo, G., Graziosi, C., Demarest, J.F., et al. HIV infection is active and progressive in lymphoid tissue during the clinically latent stage of disease. Nature 362:355, 1993.
2. Wei, X., Ghosh, S.K., Taylor, M.E., et al. Viral dynamics in human immunodeficiency virus type 1 infection. Nature 373:117, 1995.
3. Ho, D.D., Neumann, A.U., Perelson, A.S., et al. Rapid turnover of plasma virions and CD4 lymphocytes in HIV-1 infection. Nature 373:123, 1995.
4. Perelson, A.S., Neumann, A.U., Markowitz, M., et al. HIV-1 dynamics in vivo: Virion clearance rate, infected cell lifespan, and viral generation time. Science 271:1582, 1996.
5. Yu, M., Poeschila, E. & Wong-Staal, F. Progress towards gene therapy for HIV infection. Gene Ther. 1:13, 1994.
6. Bridges, S.H. & Sarver, N. Gene therapy and immune restoration for HIV disease. Lancet 345:427, 1995.
7. Poeschla, E., Corbeau, P. & Wong-Staal, F. Development of HIV vectors for anti-HIV gene therapy. Proc. Natl. Acad. Sci. USA 93:11395, 1996.
8. Lori, F., Lisziewicz, J., Smythe, J., et al. Rapid protection against human immunodeficiency virus type 1 (HIV-1) replication mediated by high efficiency non-retroviral delivery of genes interfering with HIV-1 tat and gag. Gene Ther. 1:27, 1994.
9. Woffendin, C., Yang, Z.-Y., Kumar, U., et al. Nonviral and viral delivery of a human immunodeficiency virus protective into primary human T cells. Proc. Natl. Acad. Sci. USA 91:11581, 1994.
10. Duan, L., Zhang, H., Oakes, J.W., et al. Molecular and virological effects of intracellular anti-Rev single-chain variable fragments on the expression of various human immunodeficiency virus-1 strains. Hum. Gene Ther. 5:1315, 1994.
11. Chen, S.-Y., Khouri, Y. & Marasco, W.A. Design of a genetic immunotoxin to eliminate toxin immunogenicity. Gene Ther. 2:116, 1995.
12. Bahner, I., Kearns, K., Hao, Q.-L., et al. Transduction of human CD34+ hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits human immunodeficiency virus type 1 replication in myelomonocytic cells produced in long-term culture. J. Virol. 70:4352, 1996.
13. Leavitt, M.C., Yu, M., Wong-Staal, F., et al. Ex vivo transduction and expansion of CD4+ lymphocytes from HIV+ donors: Prelude to a ribozyme gene therapy trial. Gene Ther. 3:599, 1996.
14. Moore, M.A.S. Ex vivo expansion and gene therapy using cord blood CD34+ cells. J. Hematother. 2:221, 1993.
15. Shpall, E.J., Jones, R.B., Bearman, S.J., et al. Transplantation of enriched CD34-positive autologous marrow into breast cancer patients following high-dose chemotherapy: Influence of CD34-positive peripheral-blood progenitors and growth factors on engraftment. J. Clin. Oncol. 12:28, 1994.
16. + cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML. Blood 83:3068, 1994.
17. Kohn, D.B., Weinberg, K.I., Nolta, J.A., et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Med. 1:1017, 1995.
18. Dunbar, C.E., Cottler-Fox, M., O'Shaughnessy, J.A., et al. Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85:3048, 1995.
19. Wagner, J.E., Rosenthal, J., Sweetman, R., et al. Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: Analysis of engraftment and acute graft-versus-host disease. Blood 88:795, 1996.
20. Kurtzberg, J., Laughlin, M., Graham, M.L., et al. Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N. Engl. J. Med. 335:157, 1996.
21. LaPorte, J.-P., Gorin, N.-C., Rubinstein, P., et al. Cord-blood transplantation from an unrelated donor in an adult with chronic myelogenous leukemia. N. Engl. J. Med. 335:167, 1996.
22. Molina, J.-M., Scadden, D.T., Sakaguchi, M., et al. Lack of evidence for infection of or effect on growth of hematopoietic progenitor cells after in vivo or in vitro exposure to human immunodeficiency virus. Blood 76:2476, 1990.
23. Kaushal, S., LaRussa, V.F., Gartner, S., et al. Exposure of human CD34+ cells to human immunodeficiency virus type 1 does not influence their expansion and proliferation of hematopoietic progenitors in vitro. Blood 88:130, 1996.
24. Junker, U., Moon, J.J., Kalfoglou, C.S., et al. Hematopoietic potential and retroviral transduction of CD34+ Thy1+ peripheral blood stem cells from asymptomatic human immunodeficiency virus type-1-infected individuals mobilized with granulocyte colony-stimulating factor. Blood 89:4299, 1997.
25. Kearns, K., Bahner, I., Bauer, G., et al. Suitability of bone marrow from HIV-1 infected donors for retrovirus-mediated gene transfer. Hum. Gene Ther. 8:301, 1997.
26. von Laer, D., Hufert, F.T., Fenner, T.E., et al. CD34+ hematopoietic progenitor cells are not a major reservoir of the human immunodeficiency virus. Blood 76:1281, 1990.
27. Davis, B.R., Schwartz, D.H., Marx, J.C., et al. Absent or rare human immunodeficiency virus infection of bone marrow stem/progenitor cells in vivo. J. Virol. 65:1985, 1991.
28. De Luca, A., Teofili, L., Antinori, A., et al. Haemopoietic CD34+ progenitor cells are not infected by HIV-1 in vivo but show impaired clonogenesis. Br. J. Haematol. 85:20, 1993.
29. Marandin, A., Katz, A., Oksenhendler, E., et al. Loss of primitive hematopoietic progenitors in patients with human immunodeficiency virus infection. Blood 88:4568, 1996.
30. Neal, T.F., Holland, H.K., Baum, C.M., et al. CD34+progenitor cells from asymptomatic patients are not a major reservoir for human immunodeficiency virus-1. Blood 86:1749, 1996.
31. Banda, N.K., Simon, G.R., Sipple, J., et al. Depletion of CD34+CD4+ cells in bone marrows from HIV-infected individuals with AIDS. Submitted.
32. McCune, J., Namikawa, R., Kaneshima, H., et al. The SCID-hu mouse: Murine model for the analysis of human hematolymphoid differentiation and function. Science 241:1632, 1988.
33. Aldrovandi, G.M., Feuer, G., Gao, L., et al. HIV-1 infection of the SCID-hu mouse: An animal model for virus pathogenesis. Nature 363:732, 1993.
34. Akkina, R.K, Rosenblatt, J.D., Campbell, A.G., et al. Modeling human lymphoid precursor cell gene therapy in the SCID-hu mouse. Blood 84:1393, 1994.
35. Collier, R.J. & Kandel, J. Structure and activity of diphtheria toxin. I. Thiol-dependent dissociation of a fraction of toxin into enzymically active and inactive fragments. J. Biol. Chem. 246:1485, 1971.
36. Harrison, G.S., Maxwell, F., Long, C.J., et al. Activation of a diphtheria toxin A gene by expression of human immunodeficiency virus-1 Tat and Rev proteins in transfected cells. Hum. Gene Ther. 2:53, 1991.
37. Harrison, G.S., Long, C.J., Curiel, T.J., et al. Inhibition of human immunodeficiency virus-1 production resulting from transduction with a retrovirus containing an HIV-regulated diphtheria toxin A chain gene. Hum. Gene Ther. 3:461, 1992.
38. Harrison, G.S., Long, C.J., Maxwell, F., et al. Inhibition of HIV production in cells containing an integrated, HIV-regulated diphtheria toxin A chain gene. AIDS Res. Hum. Retrovir. 8:39, 1992.
39. Curiel, T.J., Nicot, D., Wang, Y., et al. Long-term inhibition of clinical and laboratory HIV isolates in human T cells containing an HIV-regulated diphtheria toxin A chain gene. Hum. Gene Ther. 4:741, 1993.
40. Dinges, M.M., Cook, D.R., King, J., et al. HIV-regulated diphtheria toxin A chain gene confers long-term protection against HIV type 1 infection in the human promonocytic cell line U937. Hum. Gene Ther. 6:1437, 1995.
41. Bonyhadi, M.L., Rabin, L., Salimi, S., et al. HIV induces thymus depletion in vivo. Nature 363:728, 1993.
42. Stanley, S.K., McCune, J.M., Kaneshima, H., et al. Human immunodeficiency virus infection of the human thymus and disruption of the thymic microenvironment in the SCID-hu mouse. J. Exp. Med. 178:1151, 1993.
43. Kollman, T.R., Pettoello-Mantovani, M., Zhuang, X., et al. Disseminated human immunodeficiency virus 1 (HIV-1) infection in SCID-hu mice after peripheral inoculation with HIV-1. J. Exp. Med. 179:513, 1994.
44. Kollman, T.R., Kim, A., Pettoello-Mantovani, M., et al. Divergent effects of chronic HIV-1 infection of human thymocyte maturation in SCID-hu mouse. J. Immunol. 154:907, 1995.
45. Broxmeyer, H.E., Kurtzerg, J., Gluckman, E., et al. Umbilical cord blood hematopoietic stem and repopulating cells in human clinical transplantation. Blood Cells 17:313, 1991.
46. Migliaccio, G., Migliaccio, A.R., Druzin, M.L., et al. Long-term generation of colony-forming cells in liquid culture of CD34[+t] cord blood cells in the presence of recombinant human stem cell factor. Blood 79:2620, 1992.
47. Lu, L., Xiao, M., Shen, R.N., et al. Enrichment, characterization, and responsiveness of single primitive CD34+ human umbilical cord blood hematopoietic progenitors with high proliferative and replating potential. Blood 81:41, 1993.
48. Cardoso, A.A., Li, M., Batard, P., et al. Human umbilical cord blood CD34+ cell purification with high yield of early progenitors. J. Hematother. 2:275, 1993.
49. Vormoor, J., Lapidot, T., Pflumio, F., et al. Immature human cord blood progenitors engraft and proliferate to high levels in severe combined immunodeficient mice. Blood 83:2489, 1994.
50. Hans, C.S., Dugan, M.J., Verfaille, C.M., et al. In vitro expansion of umbilical cord blood committed and primitive hematopoietic progenitors. Exp. Hematol. 22:723, 1994.
51. Yu, M., Leavitt, M.C., Maruyama, M., et al. Intracellular immunization of human fetal cord blood stem/progenitor cells with a ribozyme against human immunodeficiency virus type 1. Proc. Natl. Acad. Sci. USA 92:699, 1995.
52. LaRochelle, A., Vormoor, J., Hanenberg, H., et al. Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: Implications for gene therapy. Nature Med. 2:1329, 1996.
53. Gluckman, E., Broxmeyer, H.E., Auerbach, A.D., et al. Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. N. Engl. J. Med. 321:1174, 1989.
54. Wagner, J.E., Kernan, N.A., Steinbuch, M., et al. Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and nonmalignant disease. Lancet 346:214, 1995.
55. Miller, A.D. & Buttimore, C. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol. Cell. Biol. 6:2895, 1986.
56. Miller, A.D. & Rosman G.J. Improved retroviral vectors for gene transfer and expression. BioTechniques 7:980, 1989.
57. Kotani, H., Newton, P.B., Zhang, S., et al. Improved methods of retroviral vector transduction and production for gene therapy. Hum. Gene Ther. 5:19, 1994.
58. Shein, H.M. & Enders, J.F. Multiplication and cytopathogenicity of simian vacuolating virus 40 in cultures of human tissues. Proc. Soc. Exp. Biol. Med. 109:495, 1962.
59. Miltenyi, S., Muller, W., Weichel, W., et al. High-gradient magnetic cell separation with MACS. Cytometry 11:231, 1990.
60. Harrison, G.S., Wang, Y., Tomczak, J., et al. Optimization of gene transfer using cationic lipids in cell lines and primary human CD4+ and CD34+ hematopoietic cells. BioTechniques 19:816, 1995.
61. Danilovs, J., Terasaki, P.I., Parks, M.S., et al. B lymphocyte isolation by thrombin nylon wool. In Tissue Culture Typing Laboratory, ed. Teraskis, P.I. UCLA, Los Angeles, 1980, p. 287.
62. Banda, N.K., Tomczak, J.A., Shpall, E.J., et al. HIV-gp120 induced cell death in hematopoietic progenitor CD34+ cells. Apoptosis 2:61, 1997.
63. Adachi, A., Gendelman, H.E., Koenig, S., et al. Production of acquired immunodeficiency syndrome-associated retrovirus in human and nonhuman cells transfected with an infectious molecular clone. J. Virol. 59:284, 1986.
64. Gartner, S., Marzovits, P., Markovitz, D.M., et al. The role of mononuclear phagocytes in HTLV-III/LAV infection. Science 223:215, 1986.
65. DiGiusto, D., Lee, R., Moon, J., et al. Hematopoietic potential of cryopreserved and ex vivo manipulated umbilical cord blood progenitor cells evaluated in vitro and in vivo. Blood 87:1261, 1996.
66. Yurasov, S., Kollmann, T.R., Kim, A., et al. Severe combined immunodeficiency mice engrafted with human T cells, B cells, and myeloid cells after transplantation with human fetal bone marrow or liver cells and implanted with human fetal thymus: A model for studying human gene therapy. Blood 89:1800, 1997.
67. Yamaizumi, M., Mekada, E., Uchida, T., et al. One molecule of diphtheria toxin fragment A introduced into the cell can kill the cell. Cell 15:245, 1978.