Lentiviral vectors for enhanced gene expression in human hematopoietic cells

Molecular Therapy

Volumn 2, Issue 5, 2000, Pages 458-469

  Ramezani, Ali ^a   Hawley, Teresa S ^a   Hawley, Robert G ^a,b  

^a HOLLAND LABORATORY   (United States)

^b GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALOCASIA MACRORRHIZOS; CUCUMBER MOSAIC VIRUS; GALLUS GALLUS; GIBBON APE LEUKEMIA VIRUS; HUMAN HERPESVIRUS 5; HUMAN IMMUNODEFICIENCY VIRUS 1; HYLOBATES SP.; MARMOTA MONAX; MURINAE; STRAWBERRY CRINKLE VIRUS; WOODCHUCK HEPATITIS B VIRUS;

CD34 ANTIGEN; DYES, REAGENTS, INDICATORS, MARKERS AND BUFFERS; GREEN FLUORESCENT PROTEIN; PHOTOPROTEIN;

ARTICLE; CELL CULTURE; CELL LINE; FETUS BLOOD; GENE EXPRESSION; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRINAE; METABOLISM; PROMOTER REGION; TRANSGENE;

ANTIGENS, CD34; CELL LINE; FETAL BLOOD; GENE EXPRESSION; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELLS; HIV-1; HUMANS; INDICATORS AND REAGENTS; LENTIVIRUS; LUMINESCENT PROTEINS; PROMOTER REGIONS (GENETICS); TRANSDUCTION, GENETIC; TRANSGENES; TUMOR CELLS, CULTURED;

EID: 0034322159     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0190     Document Type: Article

References (74)

1. Naldini, L., et al. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263-267.
2. Naldini, L., Blomer, 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.
3. Kafri, T., Blomer, U., Peterson, D. A., Gage, F. H., and Verma, I. M. (1997). Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat. Genet. 17: 314-317.
4. Miyoshi, H., Takahashi, M., Gage, F. H., and Verma, I. M. (1997). Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc. Natl. Acad. Sci. USA 94: 10319-10323.
5. Zennou, V., Petit, C., Guetard, D., Nerhbass, U., Montagnier, L., and Charneau, P. (2000). HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101: 173-185.
6. Cheshier, S. H., Morrison, S. J., Liao, X., and Weissman, I. L. (1999). In vivo proliferation and cell cycle kinetics of long-term self-renewing hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 96: 3120-3125.
7. + cells in non-obese diabetic/severe combined immune-deficient mice. Blood 92: 2641-2649.
8. + cells with a human immunodeficiency virus type 1-based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G. J. Virol. 70: 2581-2585.
9. Reiser, J., Harmison, G., Kluepfel-Stahl, S., Brady, R. O., Karlsson, S., and Schubert, M. (1996). Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles. Proc. Natl. Acad. Sci. USA 93: 15266-15271.
10. 1 human hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 95: 11939-11944.
11. - human hematopoietic cells by HIV-1-based lentiviral vectors. Proc. Natl. Acad. Sci. USA 96: 2988-2993.
12. Sutton, R. E., Reitsma, M. J., Uchida, N., and Brown, P. O. (1999). Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent. J. Virol. 73: 3649-3660.
13. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283: 682-686.
14. + cells. J. Virol. 74: 1286-1295.
15. Boshart, M., Weber, F., Jahn, G., Dorsch-Hasler, K., Fleckenstein, B., and Schaffner, W. (1985). A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus. Cell 41: 521-530.
16. Baskar, J. F., et al. (1996). The enhancer domain of the human major immediate-early promoter determines cell type-specific expression in transgenic mice. J. Virol. 70: 3207-3214.
17. Scharfmann, R., Axelrod, J. H., and Verma, I. M. (1991). Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc. Natl. Acad. Sci. USA 88: 4626-4630.
18. Kay, M. A., et al. (1992). Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc. Natl. Acad. Sci. USA 89: 89-93.
19. Miyoshi, H., Blomer, U., Takahashi, M., Gage, F. H., and Verma, I. M. (1998). Development of a self-in activating lentivirus vector. J. Virol. 72: 8150-8157.
20. Zufferey, R., et al. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72: 9873-9880.
21. Cheng, L., et al. (1997). A GFP reporter system to assess gene transfer and expression in viable human hematopoietic progenitors. Gene Ther. 4: 1013-1022.
22. Hawley, R. G., Lieu, F. H. L., Fong, A. Z. C., and Hawley, T. S. (1994). Versatile retroviral vectors for potential use in gene therapy. Gene Ther. 1: 136-138.
23. Eglitis, M. A., Schneiderman, R. D., Rice, P. M., and Eiden, M. V. (1995). Evaluation of retroviral vectors based on the gibbon ape leukemia virus. Gene Ther. 2: 486-492.
24. Kim, D. W., Uetsuki, T., Kaziro, Y., Yamaguchi, N., and Sugano, S. (1990). Use of the human elongation factor 1 alpha promoter as a versatile and efficient expression system. Gene 91: 217-223.
25. Niwa, H., Yamamura, K., and Miyazaki, J. (1991). Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108: 193-199.
26. Lim, B., Apperley, J. F., Orkin, S. H., and Williams, D. A. (1989). Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 86: 8892-8896.
27. Hawley, R. G., et al. (1996). Thrombopoietic potential and serial repopulating ability of murine hematopoietic stem cells constitutively expressing interleukin-11. Proc. Natl. Acad. Sci. USA 93: 10297-10302.
28. Pawliuk, R., Eaves, C. J., and Humphries, R. K. (1997). Sustained high-level reconstitution of the hematopoietic system by preselected hematopoietic cells expressing a transduced cell-surface antigen. Hum. Gene Ther. 8: 1595-1604.
29. Conneally, E., Eaves, C. J., and Humphries, R. K. (1998). Efficient retroviral-mediated gene transfer to human cord blood stem cells with in vivo repopulating potential. Blood 91: 3487-3493.
30. Cheng, L., et al. (1998). Sustained gene expression in retrovirally transduced, engrafting human hematopoietic stem cells and their lympho-myeloid progeny. Blood 92: 83-92.
31. + cells expands progenitor cell numbers, preserves engraftment capacity in nonobese diabetic/severe combined immunodeficient mice, and enhances retroviral transduction efficiency. Hum. Gene Ther. 10:2927-2940.
32. - cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: Dissociation of SRC phenotype and function. Blood 95: 102-110.
33. Kawakami, T. G., Huff, S. G., Buckley, P. M., Dungworth, D. L., Synder, S. P., and Gilden, R. V. (1972). C-type virus associated with gibbon lymphosarcoma. Nat. New Biol. 235: 170-171.
34. Hilberg, F., Stocking, C., Ostertag, W., and Grez, M. (1987). Functional analysis of a retroviral host-range mutant: altered long terminal repeat sequences allow expression in embryonal carcinoma cells. Proc. Natl. Acad. Sci. USA 84: 5232-5236.
35. Uetsuki, T., Naiot, A., Nagata, S., and Kaziro, Y. (1989). Isolation and characterization of the human chromosomal gene for polypeptide chain elongation factor-1 alpha. J. Biol. Chem. 264: 5791-5798.
36. Taboit-Dameron, F., et al. (1999). Association of the 5′HS4 sequence of the chicken β-globin locus control region with human EF1 α gene promoter induces ubiquitous and high expression of human CD55 and CD59 cDNAs in transgenic rabbits. Transgenic Res. 8: 223-235.
37. + cells using particle-mediated gene delivery. Hum. Gene Ther. 9: 2197-2205.
38. Chang, L.-J., Urlacher, V., Iwakuma, T., Cui, Y., and Zucali, J. (1999). Efficacy and safety analyses of a recombinant human immunodeficiency virus type 1 derived vector system. Gene Ther. 6: 715-728.
39. Araki, K., Imaizumi, T., Ikuyama, K., Oike, Y., and Yamamura, K. (1997). Efficiency of recombination by Cre transient expression in embryonic stem cells: comparison of various promoters. J. Biochem. 122: 977-982.
40. Okabe, M., Ikawa, M., Kiminami, K., Nakanishi, T., and Nishimune, Y. (1997). 'Green mice' as a source of ubiquitous green cells. FEBS Lett. 407: 313-319.
41. Shimitohno, K., and Temin, H. M. (1982). Loss of intervening sequences in genomic mouse alpha-globin DNA inserted in an infectious retrovirus vector. Nature 299: 265-268.
42. Malim, M. H., Hauber, J., Le, S. Y., Maizel, J. V., and Cullen, B. R. (1989). The HIV-1 rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA. Nature 338: 254-257.
43. Zufferey, R., Donello, J. E., Trono, D., and Hope, T. J. (1999). Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73: 2886-2892.
44. Civin, C., Strauss, L. C., Brovall, C., Fackler, M. J., Schwartz, J. F., and Shaper, J. H. (1984). Antigenic analysis of haematopoiesis. III. A haematopoietic progenitor cell surface antigen defined by a monoclonal antibody raised against KG1a cells. J. Immunol. 133: 157-165.
45. Furley, A. J., et al. (1986). Divergent molecular phenotypes of KG1 and KG1 a myeloid cell lines. Blood 68: 1101-1107.
46. Flanagan, J. R., Krieg, A. M., Max, E. E., and Khan, A. S. (1989). Negative control region at the 5′ end of murine leukemia virus long terminal repeats. MoI. Cell. Biol. 9: 739-746.
47. Mizushima, S., and Nagata, S. (1990). pEF-BOS, a powerful mammalian expression vector. Nucleic Acids Res. 18: 5322.
48. Singer-Sam, J., et al. (1984). Sequence of the promoter region of the gene for human X-linked 3 phosphoglycerate kinase. Gene 32: 409-417.
49. Rasheed, S., Nelson-Rees, W. A., Toth, E. M., Arnstein, P., and Gardner, M. B. (1974). Characterization of a newly derived human sarcoma cell line (HT-1080). Cancer 33: 1027-1033.
50. DuBridge, R. B., Tang, P., Hsia, H. C., Leong, P. M., Miller, J. H., and Calos, M. P. (1987). Analysis of mutation in human cells by using an Epstein-Barr virus shuttle system. Mol. Cell. Biol. 7: 379-387.
51. Koeffler, H. P., Billing, R., Lusis, A. J., Sparkes, R., and Golde, D. W. (1980). An undifferentiated variant derived from the human acute myelogenous leukemia cell line (KG-1). Blood 56: 265-273.
52. Gervaix, A., West, D., Leoni, L. M., Richman, D. D., Wong-Staal, F., and Corbeil, J. (1997). A new reporter cell line to monitor HIV infection and drug susceptibility in vitro. Proc. Natl. Acad. Sci. USA 94: 4653-4658.
53. Hawley, T. S., Sabourin, L. A., and Hawley, R. G. (1989). Comparative analysis of retroviral vector expression in mouse embryonal carcinoma cells. Plasmid 22: 120-131.
54. Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L., and Trono, D. (1997). Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotech. 15: 871-875.
55. Bahnson, A. B., et al. (1995). Centrifugal enhancement of retroviral mediated gene transfer. J. Virol. Methods 54: 131-143.
56. Cheng, L., Fu, J., Tsukamoto, A., and Hawley, R. G. (1996). Use of green fluorescent protein (GFP) variants to monitor gene transfer and expression in mammalian cells. Nat. Biotech. 14: 606-609.
57. Ausubel, F. M., et al. (1999). Short Protocols in Molecular Biology. Wiley, New York.
58. Tsujimoto, Y., Bashir, M. M., Givol, I., Cossman, J., Jaffe, E., and Croce, C. M. (1987). DNA rearrangements in human follicular lymphoma can involve the 5′ or the 3′ region of the bcl-2 gene. Proc. Natl. Acad. Sci. USA 84: 1329-1331.
59. Hawley, R. G., Covarrubias, L., Hawley, T., and Mintz, B. (1987). Handicapped retroviral vectors efficiently transduce foreign genes into hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 84: 2406-2410.
60. Cupelli, L., Okenquist, S. A., Trubetskoy, A., and Lenz, J. (1998). The secondary structure of the R region of a murine leukemia virus is important for stimulation of long terminal repeat-driven gene expression. J. Virol. 72: 7807-7814.
61. Quinn, J. P., Holbrook, N., and Levens, D. (1987). Binding of a cellular protein to the gibbon ape leukemia virus enhancer. Mol. Cell. Biol. 7: 2735-2744.
62. Trainor, C., Scott, M. L., Josephs, S. F., Fry, K. E., and Reitz, M. S., Jr. (1984). Nucleotide sequence of the large terminal repeat of two different strains of gibbon ape leukemia virus. Virology 137: 201-205.
63. Dull, T., et al. (1998). A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72: 8463-8471.
64. Fahlman, C., et al. (1999). Lentiviral gene transfer into secondary NODSCID repopulating cells and lentiviral vector design for expression in CD34+ cells. Blood 94(Suppl. 1): 359a.
65. Graham, F. L., Smiley, J., Russell, W. C., and Nairn, R. (1977). Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36: 59-74.
66. Gorman, C. M., Gies, D., McCray, G., and Huang, M. (1989). The human cytomegalovirus major immediate early promoter can be trans-activated by adenovirus early proteins. Virology 171: 377-385.
67. King, J. A., et al. (1998). The extended packaging sequence of MoMLV contains a constitutive mRNA nuclear export function. FEBS Lett. 434: 367-371.
68. Kung, S. M. P., An, D. S., and Chen, I. S. Y. (2000). A murine leukemia virus (MuLV) long terminal repeat derived from rhesus macaques in the context of a lentivirus vector and MuLV gag sequence results in high-level gene expression in human T lymphocytes. J. Virol. 74: 3668-3681.
69. Zaiss, A.-K., and Chang, L.-J. (2000). Differential regulation of retroviral and lentiviral transgene expression. Mol. Ther. 1: S307.
70. Gallardo, H. F., Tan, C., Ory, D., and Sadelain, M. (1997). Recombinant retroviruses pseudotyped with the vesicular stomatitis virus G glycoprotein mediate both stable gene transfer and pseudotransduction in human peripheral blood lymphocytes. Blood 90: 952-957.
71. + cells with HIV-1-derived lentiviral vectors. Mol. Ther. 2: 71-80.
72. Loeb, J., Harris, M., and Hope, T. (2000). The woodchuck hepatitis virus posttranscriptional regulatory element increases transgene expression by enhancing the 3′-end metabolism of mRNAs. Mol. Ther. 1: S142.
73. Minvielle-Sebastia, L., and Keller, W. (1999). mRNA polyadenylation and its coupling to other RNA processing reactions and to transcription. Curr. Opin. Cell Biol. 11: 352-357.
74. Cui, Y., Iwakuma, T., and Chang, L.-J. (1999). Contributions of viral splice sites and cis-regulatory elements to lentivirus vector function. J. Virol. 73: 6171-6176.