RevM10-expressing T cells derived in vivo from transduced human hematopoietic stem-progenitor cells inhibit human immunodeficiency virus replication

Journal of Virology

Volumn 71, Issue 6, 1997, Pages 4707-4716

  Bonyhadi, Mark L ^a   Moss, Katherine ^a   Voytovich, Amy ^a   Auten, Jennifer ^a   Kalfoglou, Creton ^a   Plavec, Ivan ^a   Forestell, Sean ^a   Su, Lishan ^a,b   Böhnlein, Ernst ^a   Kaneshima, Hideto ^a  

^a NOVARTIS PHARMA AG   (Switzerland)

^b UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MUTANT PROTEIN; REV PROTEIN;

ANIMAL CELL; ANIMAL MODEL; ARTICLE; GENE EXPRESSION; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; MOUSE; NONHUMAN; PRIORITY JOURNAL; T LYMPHOCYTE; TRANSGENE; VIRUS INHIBITION; VIRUS REPLICATION;

EID: 0030919846     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/jvi.71.6.4707-4716.1997     Document Type: Article

References (43)

1. Aldrovandi, G. M., G. Feuer, L. Gao, B. Jamieson, M. Kristeva, I. S. Chen, and J. A. Zack. 1993. The SCID-hu mouse as a model for HIV-1 infection. Nature 363:732-736.
2. Bahnson, A. B., J. T. Dunigan, B. E. Baysal, T. Mohney, R. W. Atchison, M. T. Nimgaonkar, E. D. Ball, and J. A. Barranger. 1995. Centrifugal enhancement of retroviral mediated gene transfer. J. Virol. Methods 54:131-143.
3. Bevec, D., M. Dobrovnik, J. Hauber, and E. Böhnlein. 1992. Inhibition of human immunodeficiency virus type 1 replication in human T cells by retroviral-mediated gene transfer of a dominant-negative rev trans-activator. Proc. Natl. Acad. Sci. USA 89:9870-9874.
4. Bodine, D. M., T. Moritz, R. E. Donahue, B. D. Luskey, S. W. Kessler, D. I. Martin, S. H. Orkin, A. W. Nienhuis, and D. A. Williams. 1993. Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Blood 82:1975-1980.
5. Bonyhadi, M. L., L. Rabin, S. Salimi, D. A. Brown, J. Kosek, J. M. McCune, and H. Kaneshima. 1993. HIV induces thymus depletion in vivo. Nature 363:728-732.
6. Bordignon, C., L. D. Notarangelo, N. Nobili, G. Ferrari, G. Casorati, P. Panina, E. Mazzolari, D. Maggioni, C. Rossi, P. Servida, A. G. Ugazio, and F. Mavilio. 1995. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270:470-475.
7. Bridges, S. H., and N. Sarver. 1995. Gene therapy and immune restoration for HIV disease. Lancet 345:427-432.
8. Challita, P.-M., and D. B. Kohn. 1994. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc. Natl. Acad. Sci. USA 91:2567-2571.
9. Champseix, C., V. Marechal, I. Khazaal, O. Schwartz, S. Fournier, N. Schlegel, G. Dranoff, O. Danos, P. Blot, E. Vilmer, J. M. Heard, B. Peault, and P. Lehn. 1996. A cell surface marker gene transferred with a retroviral vector into CD34+ cord blood cells is expressed by their T-cell progeny in the SCID-hu thymus. Blood 88:107-113.
10. Conneally, E., P. Bardy, C. J. Eaves, T. Thomas, S. Chappel, E. J. Shpall, and R. K. Humphries. 1996. Rapid and efficient selection of human hematopoietic cells expressing murine heat-stable antigen as an indicator of retroviral-mediated gene transfer. Blood 87:456-464.
11. DiGiusto, D. L., R. Lee, J. Moon, K. Moss, T. O'Toole, A. Voytovich, D. Webster, and J. J. Mule. 1996. Hematopoietic potential of cryopreserved and ex vivo manipulated umbilical cord blood progenitor cells evaluated in vitro and in vivo. Blood 87:1261-1271.
12. Dunbar, C. E., M. Cottler-Fox, J. A. O'Shaughnessy, S. Doren, C. Carter, R. Berenson, S. Brown, R. C. Moen, J. Greenblatt, F. M. Stewart, S. F. Leitman, W. H. Wilson, K. Cowan, N. S. Young, and A. W. Nienhuis. 1995. Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85:3048-3057.
13. Embretson, J., M. Zupancic, J. L. Ribas, A. Burke, P. Racz, K. Tenner-Racz, and A. T. Haase. 1993. Massive covert infection of helper T lymphocytes and macrophages by HIV during the incubation period of AIDS. Nature 362: 359-362.
14. Escaich, S., C. Kalfoglou, I. Plavec, S. Kaushal, J. D. Mosca, and E. Böhnlein. 1995. RevM10-mediated inhibition of HIV-1 replication in chronically infected T cells. Hum. Genet. Ther. 6:625-634.
15. Fauci, A. S. 1993. Multifactorial nature of human immunodeficiency virus disease: implications for therapy. Science 262:1011-1018.
16. Forestell, S. P., E. Böhnlein, and R. J. Rigg. 1995. Retroviral end-point titer is not predictive of gene transfer efficiency: implications for vector production. Gene Ther. 2:723-730.
17. Fox, B. A., C. Woffendin, Z.-Y. Yang, H. San, U. Ranga, D. Gordon, J. Osterholzer, and G. J. Nabel. 1995. Genetic modification of human peripheral blood lymphocytes with a transdominant negative form of Rev: safety and toxicity. Hum. Genet. Ther. 6:997-1004.
18. Haapala, D. K., W. G. Robey, S. D. Oroszlan, and W. P. Tsai. 1985. Isolation from cats of an endogenous type C virus with a novel envelope glycoprotein. J. Virol. 53:827-833.
19. Harper, M. E., L. M. Marselle, R. C. Gallo, and F. Wong-Staal. 1986. Detection of lymphocytes expressing human T lymphotropic virus type III in lymph nodes and peripheral blood from infected individuals by in situ hybridization. Proc. Natl. Acad. Sci. USA 83:772-776.
20. Ho, D. D., T. R. Rota, and M. S. Hirsch. 1986. Infection of monocyte/ macrophages by human T lymphotropic virus type III. J. Clin. Invest. 77: 1712-1715.
21. Jang, S. K., M. V. Davies, R. J. Kaufman, and E. Wimmer. 1989. Initiation of protein synthesis by internal entry of ribosomes into 5′ nontranslated region of encephalomyocarditis virus RNA in vivo. J. Virol. 63:1651-1660.
22. Kohn, D. B., K. I. Weinberg, J. A. Nolta, L. N. Heiss, C. Lenarsky, G. M. Crooks, M. E. Hanley, G. Annett, J. S. Brooks, A. El-Khoureiy, K. Lawrence, S. Wells, R. C. Moen, J. Bastian, D. E. Williams-Herman, M. Elder, D. Wara, T. Bowen, M. S. Hershfield, C. A. Mullen, R. M. Blaese, and R. Parkman. 1996. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Med. 1:1017-1023.
23. Macatonia, S. E., R. Lau, S. Patterson, A. J. Pinching, and S. C. Knight. 1990. Dendritic cell infection, depletion and dysfunction in HIV-infected individuals. Immunology 71:38-45.
24. Malim, M. H., S. Böhnlein, J. Hauber, and B. R. Cullen. 1989. Functional dissection of the HIV-1 Rev trans-activator - derivation of a trans-dominant repressor of Rev function. Cell 58:205-214.
25. Malim, M. H., W. W. Freimuth, J. Liu, T. J. Boyle, H. K. Lyerly, B. R. Cullen, and G. J. Nabel. 1992. Stable expression of transdominant Rev protein in human T cells inhibits human immunodeficiency virus replication. J. Exp. Med. 176:1197-1201.
26. McCune, J. M., R. Namikawa, H. Kaneshima, L. D. Shultz, M. Lieberman, and I. L. Weissman. 1988. The SCID-hu mouse: a model for the analysis of human hematolymphoid differentiation and function. Science 241:1632-1639.
27. McCune, J., H. Kaneshima, J. Krowka, R. Namikawa, H. Outzen, B. Peault, L. Rabin, C. C. Shih, E. Yee, M. Lieberman, I. L. Weissman, and L. Shultz. 1991. The SCID-hu mouse: a small animal model for HIV infection and pathogenesis. Annu. Rev. Immunol. 9:399-429.
28. + T lymphocytes in spleens of human immunodeficiency virus-positive patients. J. Virol. 69:4737-4745.
29. Migita, M., J. A. Medin, R. Pawliuk, S. Jacobson, J. W. Nagle, S. Anderson, M. Amiri, R. K. Humphries, and S. Karlsson. 1995. Selection of transduced CD34+ progenitors and enzymatic correction of cells from Gaucher patients, with bicistronic vectors. Proc. Natl. Acad. Sci. USA 92:12075-12079.
30. Murray, L., B. Chen, A. Galy, S. Chen, R. Tushinski, N. Uchida, R. Negrin, G. Tricot, S. Jagannath, D. Vesole, B. Barlogie, R. Hoffman, and A. Tsukamoto. 1995. Enrichment of human hematopoietic stem cell activity in the CD34+Thy-1+Lin-subpopulation from mobilized peripheral blood. Blood 85:368-378.
31. Namikawa, R., K. N. Weilbaecher, H. Kaneshima, E. J. Yee, and J. M. McCune. 1990. Long-term human hematopoiesis in the SCID-hu mouse. J. Exp. Med. 172:1055-1063.
32. Nolta, J. A., M. A. Dao, S. Wells, E. M. Smogorzewska, and D. B. Kohn. 1996. Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice. Proc. Natl. Acad. Sci. USA 93:2414-2419.
33. Plavec, I., A. Voytovich, K. Moss, D. Webster, M. Hanley, S. Escaich, K. E. Ho, E. Böhnlein, and D. L. DiGiusto. 1996. Sustained retroviral gene marking and expression in lymphoid and myeloid cells derived from transduced hematopoietic progenitor cells. Gene Ther. 3:717-724.
34. Ragheb, J. A., P. Bressler, M. Daucher, L. Chiang, M. K. L. Chuah, T. Vandendriessche, and R. A. Morgan. 1995. Analysis of trans-dominant mutants of the HIV type 1 rev protein for their ability to inhibit rev function, HIV type 1 replication, and their use as anti-HIV gene therapeutics. AIDS Res. Hum. Retroviruses 11:1343-1353.
35. Riddell, S. R., M. Elliott, D. A. Lewinsohn, M. J. Gilbert, L. Wilson, S. A. Manley, S. D. Lupton, R. W. Overell, T. C. Reynolds, L. Corey, and P. D. Greenberg. 1996. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nature Med. 2:216-223.
36. Rigg, R. J., J. Chen, J. S. Dando, S. P. Forestell, I. Plavec, and E. Böhnlein. 1996. A novel human amphotropic packaging cell line: high titer, complement resistance, and improved safety. Virology 218:290-295.
37. Rigg, R. J., J. S. Dando, S. Escaich, I. Plavec, and E. Böhnlein. 1995. Detection of intracellular HIV-1 Rev protein by flow cytometry. J. Immunol. Methods 188:187-195.
38. Rosenzweig, M., D. P. Clark, and G. N. Gaulton. 1993. Selective thymocyte depletion in neonatal HIV-1 thymic infection. AIDS 7:1601-1605.
39. Su, L., H. Kaneshima, M. Bonyhadi, S. Salimi, D. Kraft, L. Rabin, and J. M. McCune. 1995. HIV-1 induced thymocyte depletion is associated with indirect cytopathicity and infection of progenitor cells in vivo. Immunity 2:25-36.
40. Tagawa, M., H. Nakauchi, L. A. Herzenberg, and G. P. Nolan. 1986. Formal proof that different-size Lyt-2 polypeptides arise from differential splicing and posttranscriptional regulation. Proc. Natl. Acad. Sci. USA 83:3422-3426.
41. Vandekerckhove, B. A., R. Namikawa, R. Bacchetta, and M. G. Roncarolo. 1992. Human hematopoietic cells and thymic epithelial cells induce tolerance via different mechanisms in the SCID-hu mouse thymus. J. Exp. Med. 175:1033-1043.
42. Woffendin, C., U. Ranga, Z. Y. Yang, L. Xu, and G. J. Nabel. 1996. Expression of a protective gene prolongs survival of T cells in human immunodeficiency virus-infected patients. Proc. Natl. Acad. Sci. USA 93:2889-2894.
43. Zucker-Franklin, D., and Y. Cao. 1989. Megakaryocytes of human immunodeficiency virus-related individuals express viral RNA. Proc. Natl. Acad. Sci. USA 86:5595-5599.