Spleen necrosis virus-derived C-type retroviral vectors for gene transfer to quiescent cells

Nature Biotechnology

Volumn 18, Issue 6, 2000, Pages 623-629

  Parveen, Zahida ^a   Krupetsky, Anna ^a   Engelstädter, Martin ^b   Cichutek, Klaus ^b   Pomerantz, Roger J ^a   Dornburg, Ralph ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

^b PAUL EHRLICH INSTITUT   (Germany)

Author keywords

Gene therapy; Gene transfer; Hematopoietic cells; Macrophages; Retrovital vectors; T lymphocytes

Indexed keywords

MATRIX PROTEIN; VIRUS VECTOR;

ANIMAL CELL; ARTICLE; GENE THERAPY; GENE TRANSFER; GENETIC ENGINEERING; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; NONHUMAN; NUCLEAR LOCALIZATION SIGNAL; PRIORITY JOURNAL; RETROVIRUS; SITE DIRECTED MUTAGENESIS; TARGET CELL; VIRUS INDUCTION; VIRUS PARTICLE;

AMINO ACID SEQUENCE; ANIMALS; BLOTTING, SOUTHERN; CELL DIVISION; CELL LINE; CELL NUCLEUS; DOGS; EXTRACELLULAR MATRIX PROTEINS; FLOW CYTOMETRY; GAMMARETROVIRUS; GENE PRODUCTS, GAG; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HELA CELLS; HUMANS; JURKAT CELLS; MACROPHAGES; MOLECULAR SEQUENCE DATA; MONOCYTES; MUTAGENESIS, SITE-DIRECTED; NECROSIS; NUCLEAR LOCALIZATION SIGNALS; PLASMIDS; SPLEEN; T-LYMPHOCYTES; TRANSFECTION; TUMOR CELLS, CULTURED;

ANIMALIA; RNA VIRUSES; SPLEEN NECROSIS VIRUS; UNIDENTIFIED RETROVIRUS;

EID: 0034045456     PISSN: 10870156     EISSN: None     Source Type: Journal    
DOI: 10.1038/76458     Document Type: Article

References (56)

1. Miller, A.D. Retrovirus packaging cells. Hum. Gene. Ther. 1, 5-14 (1990).
2. Dornburg, R. Reticuloendotheliosis viruses and derived vectors. Gene Ther. 2, 301-310 (1995).
3. Robbins, P.D., Tahara, H. & Ghivizzani, S.C. Viral vectors for gene therapy. Trends Biotechnol 16, 35-40 (1998).
4. Miller, A.D. Retroviral vectors. Curr. To.p Microbiol. Immunol. 158, 1-24 (1992).
5. Gilboa, E. & Smith, C. Gene therapy for infectious diseases: the AIDS model. Trends Genet. 10, 139-144 (1994).
6. Anderson, W.F. Human gene therapy. Nature 392, 25-30 (1998).
7. Pomerantz, R.J. & Trono, D. Genetic therapies for HIV infections: promise for the future. AIDS 9, 985-993 (1995).
8. Vile, R.G., Tuszynski, A. & Castleden, S. Retroviral vectors. from laboratory tools to molecular medicine. Mol. Biotechnol., 139-158 (1996).
9. Weichselbaum, R.R. & Kufe, D. Gene therapy of cancer. Lancet 349 (Suppl. 2), SII10-SII12 (1997).
10. Blaese, M. et al. Vectors in cancer therapy: how will they deliver?. Cancer Gene Ther. 2, 291-297 (1995).
11. Vile, R. & Russell, S.J. Gene transfer technologies for the gene therapy of cancer. Gene Ther. 1, 88-98 (1994).
12. Varmus, H.E. & Brown, P. In Mobile DNA (eds Berg, D.E. & Howe, M.M.) 53-108 (American Society for Microbiology, Washington, D.C.; 1988).
13. Temin, H. M. In Gene transfer (ed. Kucherlapati, R.) 144-187 (Plenum Press, New York; 1986).
14. Bukrinsky, M.I. et al. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365, 666-670 (1993).
15. Lewis, P. & Emerman, M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J. Virol. 68, 510-516 (1994).
16. Schwedler, U., Kornbluth, R.S. & Trono, D. 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 (1999).
17. Miyake, K., Suzuki, N., Matsuoka, H., Tohyama, T. & Shimada, T. Stable integration of human immunodeficiency virus-based retroviral vectors into the chromosomes of non-dividing cells. Hum. Gene Ther. 9, 467-475 (1999).
18. Naldini, L., Blomer, U., Gage, F.H., Trono, D. & Verma, I.M. 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 (1996).
19. Zufferey, R. et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880 (1998).
20. Dull, T. et al. A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463-8471 (1998).
21. Miyake, K., Suzuki, N., Matsuoka, H., Tohyama, T. & Shimada, T. Stable integration of human immunodeficiency virus-based retroviral vectors into the chromosomes of nondividing cells. Hum. Gene Ther. 9, 467-475 (1998).
22. Poeschla, E., Corbeau, P. & Wong-Staal, F. Development of HIV vectors for anti-HIV gene therapy. Proc. Natl. Acad. Sci. USA 93, 11395-11399 (1996).
23. Hu, W.-S. & Temin, H.M. Retroviral recombination and reverse transcription. Science 250, 1227-1233 (1990).
24. Temin, H. M. Sex and recombination in retroviruses. Trends Genet. 7, 71-74 (1991).
25. Dornburg, R. From the natural evolution to the genetic manipulation of the host range of retroviruses. Biol. Chem 378, 457-468 (1997).
26. Kewalramani, V. N., Panganiban, A. T. & Emerman, M. Spleen necrosis virus, an avian immunosuppressive retrovirus, shares a receptor with the type D simian retroviruses. J. Virol. 66, 3026-3031 (1992).
27. Martinez, I. & Dornburg, R. Mapping of receptor binding domains in the envelope protein of spleen necrosis virus. J. Virol. 69, 4339-4346 (1995).
28. Martinez, I. & Dornburg, R. Mutational analysis of the envelope protein of spleen necrosis virus. J. Virol. 70, 6036-6043 (1996).
29. Gautier, R., Jiang, A., Rousseau, V., Dornburg, R. & Jaffredo, T. The reticuloendotheliosis viruses strain A, REV-A or spleen necrosis virus, SNV, are non-infectious in human cells. J. Virol. 74, 518-522, in press (2000).
30. Chu, T.-H. & Dornburg, R. Retroviral vector particles displaying the antigen-binding site of an antibody enable cell-type-specific gene transfer. J. Virol. 69, 2659-2663 (1995).
31. Chu, T.-H., Martinez, I., Sheay, W.C. & Dornburg, R. Cell targeting with retroviral vector particles containing antibody-envelope fusion proteins. Gene. Ther. 1, 292-299 (1994).
32. Chu, T.-H. & Dornburg, R. Towards highly-efficient cell-type-specific gene transfer with retroviral vectors that display a single chain antibody. J. Virol 71, 720-725 (1997).
33. Jiang, A., Chu, T.-H. T., Nocken, F., Cichutek, K. & Dornburg, R. Cell-type-specific gene transfer into human cells with retroviral vectors that display single-chain antibodies. J. Virol. 72, 10148-10156 (1998).
34. Engelstädter, M. et al. Targeting human T-cells by retroviral vectors displaying antibody domains selected from a phage display library. Hum. Gene Ther. 11, 293-303 (2000).
35. Jiang, A. & Dornburg, R. In vivo cell-type-specific gene delivery with retroviral vectors that display single chain antibodies. Gene Ther. 6, 1982-1987 (1999).
36. Kootstra, N.A. & Schuitemaker, H. Phenotype of HIV-1 lacking a functional nuclear localization signal in matrix protein of gag and vpr is comparable to wildtype HIV-1 in primary macrophages. Virology 253, 170-180 (1999).
37. Fouchier, R.A., Meyer, B.E., Simon, J.H., Fischer, U. & Malim, M.H. HIV-1 infection of non-dividing cells: evidence that the amino-terminal basic region of the viral matrix protein is important for gag processing but not for post-entry nuclear import. EMBO J. 16, 4531-4539 (1997).
38. Yoneda, Y. How proteins are transported from cytoplasm to the nucleus. J Biochem. (Tokyo) 121, 811-817 (1997).
39. Gorlich, D. Nuclear protein import. Can. Opin. Cell Bid. 9, 412-419 (1997).
40. Moroianu, J. Molecular mechanisms of nuclear protein transport. Crit. Rev. Eukaryot. Gene Expr. 7, 61-72 (1997).
41. Boulikas, T. Nuclear import of protein kinases and cyclins. J. Cell Biochem. 60, 61-82 (1996).
42. Martinez, I. & Dornburg, R. Partial reconstitution of a replication-competent retrovirus in helper cells with partial overlaps between vector and helper cell genomes. Hum. Gene Ther. 7, 705-712 (1996).
43. Stevenson, M. & Gendelman, H.E. Cellular and viral determinants that regulate HIV-1 infection in macrophages. J. Leuk. Biol. 56, 278-288 (1994).
44. Huang, Z.B. et al. Infection of macrophages with lymphotropic human immunodeficiency virus type 1 can be arrested after viral DNA synthesis. J. Virol. 67, 6893-6896 (1993).
45. Blaese, R.M. et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270, 475-480 (1995).
46. von Schwedler, U., Kornbluth, R.S. & Trono, D. 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 (1994).
47. Collman, R. et al. Infection of monocyte-derived macrophages with human immunodeficiency virus type 1 (HIV-1). monocyte-tropic and lymphocyte-tropic strains of HIV-1 show distinctive patterns of replication in a panel of cell types. J. Exp. Med. 170, 1149-1163 (1989).
48. Temin, H.M. The protovirus hypothesis: speculations on the significance of RNA-directed DNA synthesis for normal development and for carcinogenesis. J. Natl. Cancer Inst. 46, 3-7 (1971).
49. Coffin, J.M. Genetic diversity and evolution of retroviruses. Curr. Top. Microbiol. Immunol. 176, 143-164 (1992).
50. Deminie, C.A. & Emerman, M. Functional exchange of an oncoretrovirus and a lentivirus matrix protein. J. Virol. 68, 4442-4449 (1994).
51. Deminie, C.A. & Emerman, M. Incorporation of human immunodeficiency virus type 1 gag proteins into murine leukemia virus virions. J. Virol. 67, 6499-6506 (1993).
52. Mikawa, T., Fischman, D.A., Dougherty, J.P. & Brown, A.M.C. In vivo analysis of a new lacZ retrovirus vector suitable for lineage marking in avian and other species. Exp. Cell Res. 195, 516-523 (1992).
53. Martinez, I. & Dornburg, R. Improved retroviral packaging lines derived from spleen necrosis virus. Virology 208, 234-241 (1995).
54. Urban, A., Neukirchen, S. & Jaeger, K.E. A rapid and efficient method for site-directed mutagenesis using one-step overlap extension per. Nucleic Acids Res. 25, 2227-2228 (1997).
55. Picard, V., Ersdal-Badju, E., Lu, A. & Bock, S.C. A rapid and efficient one-tube PCR-based mutagenesis technique using pfu DNA polymerase. Nucleic Acids Res. 22, 2587-2591 (1994).
56. Sambrook, J., Fritsch, E.F. & Maniatis, T. Molecular cloning: a laboratory manual. (Cold Spring Harbor Press,Cold Spring Harbor, NY: 1995).