Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

Nature Biotechnology

Volumn 15, Issue 9, 1997, Pages 871-875

  Zufferey, Romain ^a,c   Nagy, Dea ^b   Mandel, Ron J ^b   Naldini, Luigi ^b   Trono, Didier ^a,c  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b CELL GENESYS INC   (United States)

^c UNIVERSITY OF GENEVA MEDICAL SCHOOL   (Switzerland)

Author keywords

Gene therapy; HIV; Retroviral vectors

Indexed keywords

ANIMAL EXPERIMENT; ARTICLE; EXPRESSION VECTOR; GENE DELETION; GENE TARGETING; GENE THERAPY; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRINAE; MALE; NONHUMAN; PRIORITY JOURNAL; RAT; VIRUS ATTENUATION; VIRUS VIRULENCE;

ANIMALS; CELLS, CULTURED; CORPUS STRIATUM; DNA, VIRAL; GENE DELETION; GENE EXPRESSION REGULATION, VIRAL; GENE THERAPY; GENES, REPORTER; GENETIC VECTORS; HIV-1; HUMANS; MACROPHAGES; RATS; TRANSDUCTION, GENETIC; TRANSFECTION; VIRAL ENVELOPE PROTEINS;

ANIMALIA; HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS;

EID: 0030819379     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt0997-871     Document Type: Article

References (40)

1. Mulligan, R.C. 1993. The basis science of gene therapy. Science 260:926-932.
2. Crystal, R.G.1995. Transfer of genes to humans: early lessons and obstacles to success. Science 270:404-410.
3. Leiden, J.M. 1995. Gene therapy—promises, pitfalls and prognosis. N. Engl. J. Med. 333:871-873.
4. Sanders, W.R. 1995. Human gene therapy—of tortoises and hares. Nature Medicine 1:1137-1138.
5. Roe, T., Reynolds, T.C., Yu, G., and Brown, P.O. 1993. Integration of murine leukemia virus DNA depends on mitosis. EMBO J. 12:2099-2108.
6. Lewis, P.F. and Emerman, M. 1994. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J. Virol. 68:510-516.
7. Lewis, P., Hensel M., and Emerman, M. 1992. Human immunodeficiency virus infection of cell arrested in the cell cycle. EMBO J. 11:3053-3058.
8. Bukrinsky, M.I., Haggerty, S., Dempsey, M.P., Sharova, N., Adzhubei, A., Spitz, L., et al. 1993. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365:666-669.
9. Von Schwedler, U., Kornbluth, R.S., and Trono, D. 1994. The nuclear localization signal of the matrix protein of human immunodeficiency virus type 1 allows the establishment of infection in macrophages and quiescent T lymphocytes. Proc. Natl. Acad. Sci. USA 91:6992-6996.
10. Gallay, P., Stitt, V., Mundy, C., Oettinger, M., and Trono, D. 1996. Role of the karyopherin pathway in human immunodeficiency type 1 nuclear import. J. Virol. 70:1027-1032.
11. Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R.C., Gage, F.H., et al. 1996. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-267.
12. Naldini, L., Blömer, U., Gage, F.H., Trono, D. and Verma, I.M. 1996. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl Acad. Sci. USA 93:11382-11388.
13. Trono D. 1995. HIV accessory proteins: leading role for the supporting cast. Cell 82:189-192.
14. Miller, R.H. and Sarver, N. 1997. HIV accessory proteins as therapeutic targets. Nature Medicine 3:389-394.
15. gag virus-like particles from recombinant baculovirus-infected insect cells. Cell 59:103-112.
16. Heinzinger, N.K., Bukrinsky, M.I., Haggerty, S.A., Ragland, A.M., Kewalramani, V., Lee, M.A., et al. 1994. The Vpr protein of human immunodeficiency virus type 1 influences nuclear localization of viral nucleic acids in nondividing host cells. Proc Nat. Acad. Sci. USA 91:7311-7315.
17. + T lymphocytes. J. Virol. 66:6489-6495.
18. von Schwedler, U., Song, J., Aiken, C., and Trono, D. 1993. vif is crucial for human immunodeficiency virus type 1 proviral DNA synthesis in infected cells. J. Virol. 67:4945-4955.
19. Klimkait, T., Strebel, K., Hoggan, M.D., Martin, M.A., and Orenstein, J.M. 1990. The human immunodeficiency virus type 1 -specific protein vpu is required for efficient virus maturation and release. J. Virol. 64:621-629.
20. Göttlinger, H.G., Dorfman, T., Cohen, E., and Haseltine, W.A. 1993. Vpu protein of human immunodeficiency virus type 1 enhances the release of capsids produced by gag gene constructs of widely divergent retroviruses. Proc. Natl. Acad. Sci. USA 90:7381-7385.
21. Miller, M.D., Warmerdam, M.T., Gaston, I., Greene, W.C., and Feinberg, M.B. 1994. The human immunodeficiency virus-1 nef gene product: a positive factor for viral infection and replication in primary lymphocytes and macrophages. J. Exp. Med. 179:101-113.
22. Spina, C.A., Kwoh, T.J., Chowers, M.Y., Guatelli, J.C., and Richman, D.D. 1994. The importance of nef in the induction of human immunodeficiency virus type 1 replication from primary quiescent CD4 lymphocytes. J. Exp. Med. 179:115-123.
23. Aiken, C. and Trono, D. 1995. Nef stimulates human immunodeficiency virus type 1 proviral DNA synthesis. J. Virol. 69:5048-5056.
24. Schwartz, O., Marechal, V., Danos, O., and Heard, J.-M. 1995. Human immunodeficiency virus type 1 Nef increases the efficiency of reverse transcription in the infected cell. J. Virol. 69:4053-4059.
25. Chowers, M.Y., Pandori, M.W., Spina, C.A., Richman, D.D., and Guatelli, J.C. 1995. The growth advantage conferred by HIV-1 nef is determined at the level of viral DNA formation and is independent of CD4 downregulation. Virology 212:451-457.
26. Aiken, C. 1997. Pseudotyping human immunodeficiency virus type 1 (HIV-1) by the glycoprotein of vesicular stomatitis virus targets HIV-1 entry to an endocytic pathway and suppresses both the requirement for Nef and the sensitivity to cyclosporin A. J. Virol. 71:5871-5877.
27. Gallay, P., Chin, D., Hope, T.J., and Trono, D. HIV-1 infection of nondividing cells mediated through the recognition of integrase by the importin/karyopherin pathway. Proc. Natl. Acad. Sci. USA. In press.
28. Levy, D.N., Fernandes, L.S., Williams, W.V., and Weiner, D.B. 1993. Induction of cell differentiation by human immunodeficiency virus 1 vpr. Cell 72:541-550.
29. Rogel, M.E., Wu, L.I., and Emerman, M. 1995. The human immunodeficiency virus type 1 vpr gene prevents cell proliferation during chronic infection. J. Virol. 69:882-888.
30. Kirchhoff, F., Greenough, T.C., Brettler, D.B., Sullivan, J.L., and Desrosiers, R.C. 1995. Absence of intact nef sequences in a long-term survivor with nonprogressive HIV-1 infection. N. Engl. J. Med. 332:228-232.
31. Deacon, N.J., Tsykin, A., Solomon, A., Smith, K., Ludford-Menting, M., Hooker, D.J., et al. 1995. Genomic structure of an attenuated quasi species of HIV-1 from a blood transfusion donor and recipients. Science 270:988-991.
32. Kestler III, H.W., Ringler, D.J., Mori, K., Panicali, D.L., Sehgal, P.K., Daniel, M.D., et al. 1991. Importance of the nef gene for maintenance of high virus loads and for development of AIDS. Cell 65:651-662.
33. Daniel, M.D., Kirchhoff, F., Czajak, S.C., Sehgal, P.K., and Desrosiers, R.C. 1992. Protective effect of a live-attenuated SIV vaccine with a deletion in the nef gene. Science 258:1938-1941.
34. Wyand, M.S., Manson, K.H., Lackner A.A., and Desrosiers R.C. 1997. Resistance of neonatal monkeys to live attenuated vaccine strains of simian immunodeficiency virus. Nature Medicine 3:32-36.
35. Gibbs, J.S., Regier, D.A., and Desrosiers R.C. 1994. Construction and in vitro properties of SIVmac mutants with deletions in “nonessential” genes. AIDS Res. Hum. Retrovir. 10:607-616.
36. Park I.-W., Myrick K., and Sodroski, J. 1994. Effects of vif mutations on cell-free infectivity and replication of simian immunodeficiency virus. J. AIDS 7:1228-1236.
37. Lang, S.M., Strahl-Hennig, C., Coulibaly, C., Hunsmann, G., Muller, J., Muller-Hermalink, H., et al. 1993. Importance of vpr for infection of rhesus monkey with simian immunodeficiency virus. J. Virol. 67:902-912.
38. Fletcher, T.M., Brichacke, B., Sharova, N., Newman, M.A., Stivahtis, G., Sharp, P.M., et al. 1997. Nuclear import and cell cycle arrest functions of the HIV-1 Vpr protein are encoded by two separate genes in HIV-2/SIV. EMBO J. 15:6155-6165.
39. Wang, B., Ge, Y.C., Palasanthiran, P., Xiang, S., Ziegler, J., Dwyer, D.E., et al. 1996. Gene defects clustered at the C-terminus of the vpr gene of HIV-1 in long-term nonprogressing mother and child pair: in vivo evolution of vpr quasispcies in blood and plasma. Virology 223:224-232.
40. Brach, M.A., Hass, R., Sherman, M.L., Gunji, H., Weichselbaum, R., and Kufe, D. 1991. Ionizing radiation induces expression and binding activity of the Nuclear Factor κB. J. Clin. Invest 88:691-695.