Retroviral bicistronic vectors

Drug News and Perspectives

Volumn 21, Issue 9, 2008, Pages 473-480

  Pfützner, Wolfgang ^a  

^a PHILIPPS UNIVERSITY MARBURG   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

RETROVIRUS BICISTRONIC VECTOR; RETROVIRUS VECTOR; UNCLASSIFIED DRUG;

ALTERNATIVE RNA SPLICING; CANCER THERAPY; CISTRON; GENE EXPRESSION; GENE STRUCTURE; GENE TECHNOLOGY; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; HUMAN; INTERNAL RIBOSOME ENTRY SITE; NONHUMAN; REVIEW; RIBOSOME; RNA TRANSLATION; SAFETY; TARGET CELL; TRANSCRIPTION INITIATION SITE; TRANSGENE; TUMOR CELL DESTRUCTION;

ANIMALS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; RETROVIRIDAE; TRANSDUCTION, GENETIC; TRANSFECTION; TRANSGENES;

EID: 61849097214     PISSN: 02140934     EISSN: None     Source Type: Journal    
DOI: 10.1358/dnp.2008.21.9.1290817     Document Type: Review

References (87)

1. Rogers, S. and Pfuderer, P. Use of viruses as carriers of added genetic information. Nature 1968, 219(5155): 749-51.
2. Friedmann, T. The origins, evolution, and directions of human gene therapy. In: The Development of Human Gene Therapy. Friedmann, T. (Ed.). Cold Spring Harbor Laboratory Press: Cold Spring Harbor 1999, 1-20.
3. Thomas, C., Ehrhardt, A. and Kay, M. Progress and problems with the use of viral vectors for gene therapy. Nat Gen Rev 2003, 4: 346-58.
4. Martinez-Salas, E. Internal ribosome entry site biology and its use in expression vectors. Curr Opin Biotechnol 1999, 10(5): 458-64.
5. Jackson, R. and Kaminski, A. Internal initiation of translation in eukaryotes: the picornavirus paradigm and beyond. RNA 1995, 1(10): 985-1000.
6. Walker, P, Hengge, U, Udey, M, Aksentijevich, I. and Vogel, J. Viral interference during simultaneous transduction with two independent helper-free retroviral vectors. Hum Gene Ther 1996, 7(9): 1131-8.
7. Di Florio, S., Sebastian, C., Fagioli, M., DiIanni, M. and Alfonsi, D. Retrovirus-mediated transfer of herpes simplex virus thymidine kinase and enhanced green fluorescence protein genes in primary T lymphocytes. Br J Haematol 2000, 110(4): 903-6.
8. Abbate, J., Lacayo, J., Prichard, M., Pari, G. and McVoy, M. Bifunctional protein conferring enhanced green fluorescencs and puromycin resistance. Biotechniques 2001, 31(2): 336-40.
9. Chung-Faye, G., Chen, M., Green, N. et al. In vivo gene therapy for colon cancer using adenovirus-mediated transfer of the fusion gene cytosine deaminase and uracil phosphoribosyltransferase. Gene Ther 2001, 8(20): 1547-54.
10. Thomas, C. and Maule, A. Limitations on the use of fused green fluorescence protein to investigate structure-function relationships for the cauliflower mosaic virus movement protein. J Gen Virol 2000, 81(Pt. 7): 1851-5.
11. Wang, X., Zinkel, S., Polonsky, K. and Fuchs, E. Transgenic studies with a keratin promoter-driven growth hormone transgene: prospects for gene therapy. Proc Natl Acad Sci U S A 1997, 94(1): 219-26.
12. Fusseneger, M. The impact of mammalian gene regulation concepts on functional genomic research, metabolic engineering and advanced gene therapies. Biotechnol Prog 2001, 17: 1-51.
13. Bandyopadhyay, P. and Temin, H. Expression of complete chicken thymidine kinase gene inserted in a retrovirus vector. Mol Cell Biol 1984, 4(4): 749-54.
14. Nakajima, K. Ikenaka, K., Nakahira, K., Morita, N. and Mikoshiba, K. An improved retroviral vector for assying promoter activity. Analysis of promoter interference in pIP211 vector. FEBS Lett 1993, 315(2): 129-33
15. Emerman, M. and Temin, H. Genes with promoters in retroviral vectors can be independently suppressed by an epigentic mechanism. Cell 1984, 39(3 Pt. 2): 449-67.
16. Emerman, M. and Temin, H. Comparison of promoter suppression in avian and murine retrovirus vectors. Nucl Acids Res 1986, 14(23): 9381-96.
17. Amendola, M., Venneri, M., Biffi, A., Vigna, E. and Naldini, L. Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol 2005, 23(1): 108-16.
18. Bunnel, B., Morgan, R. and Adolph, K. Retrovirus-mediated gene transfer. In: Viral Genome Methods. Adolph, K.W. (Ed.). CRC Press Inc., Boca Raton, 1996: 4-23.
19. Hildinger, M., Schilz, A., Eckert, H. et al. Bicistronic vectors for combining myeloprotection with cell-surface marking. Gene Ther 1999, 6(7): 1222-30.
20. Zhu, Y. and Plannelles, V. A multigene lentiviral vector system based on differential splicing. Methods Mol Med 2003, 76: 433-48.
21. Hildinger, M., Fehse, B., Hegewisch-Becker, S., John, J., Rafferty, J., Ostertag, W. and Baum, C. Dominant selection of hhematopoietic progenitor cells with retro-viral MDR1 co-expression vectors. Hum Gene Ther 1998, 9(1): 33-42.
22. Ghattas, I., Sanes, J. and Majors, J. The encephalomyocarditis virus internal ribosome entry site allows efficient co-expression of two genes from a recombinant provirus in cultured cells and in embryos. Mol Cell Biol 1991, 11(12): 5848-59.
23. Bowtell, D., Cory, S., Johnson, G. and Gonda, T. Comparison of expression in hemopoietic cells by retroviral vectors carrying two genes. J Virol 1988, 62(7): 2464-73.
24. Pulsinelli, G.A. and Temin, H. Characterization of large deletions occurring during a single round of retrovirus vector replication: novel deletion mechanism involving errors in strand transfer. J Virol 1991, 65(9): 4786-97.
25. Szymczak, A., Workman, C., Wang, Y, Vignali, K., Dilioglou, S., Vanin, E. and Vignali, D. Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat Biotechnol 2004, 22(5): 589-94.
26. Klump, H., Schiedlmeier, B., Vogt, B., Ryan, M., Ostertag, W. and Baum, C. Retroviral-vector mediated expression of HoxB4 in hematopoietic cells using a novel coexpression strategy. Gene Ther 2001, 8(10): 811-7.
27. Donnelly, M., Luke, G., Mehrotra, A., Li, X., Hughes, L., Gani, D. and Ryan, M. Analysis of the aphthovirus 2A/2B polyprotein 'cleavage' mechanism indicates not a proteolytic reaction, but a novel translational effect: A putative ribosomal 'skip'. J Gen Virol 2001, 82(Pt. 5): 1013-25.
28. Chinnasamy, D., Milsom, M., Shaffer, J. et al. Multicistronic lentiviral vectors containing the FMDV 2A cleavage factor demonstrate robust expression of encoded genes at limiting MOI. Virology J 2006, 3: 14.
29. Bradley, J., Johnson, D. and Rubenstein, D. Molecular Medicine. Blackwell Science Ltd: Oxford 2001, 34-6.
30. Ngoi, S., Chien, A. and Lee, C. Exploiting internal ribosome entry sites in gene therapy vector design. Curr Gene Ther 2004, 4(1): 15-31.
31. Jackson, R., Howell, M. and Kaminski, A. The novel mechanism of initiation of picornavirus RNA translation. Trends Biochem Sci 1990, 15(12): 477-83.
32. Müller, P., Oumard, A., Wirth, D., Kröger, A. and Hauser, H. Polyvalent vectors for coexpression of multiple genes. In: Plasmids for Therapy and Vaccination. Schleef, M. (Ed.) Wiley-VCH, Weinheim, Germany 2001, 119-37.
33. Attal, J., Theron, M., Taboit, F., Cajero-Juarez, M., Kann, G., Bolifraud, P. and Houdebine, L. The RU5 ('R') region ftom human leukaemia viruses (HTLV-1) contains an internal ribosome entry site (IRES)-like sequence. FEBS Lett 1996, 392(3): 220-4.
34. Bieleski, L. and Talbot, S. Kaposi's sarcoma-associated herpesvirus vCyclin open reading frame contains an internal ribosome entry site. J Virol 2001, 75(4): 1864-9.
35. Nanbru, C., Lafon, I., Audigier, S., Gensac, M., Vagner, S., Huez, G. and Prats, A. Alternative translation of the proto-oncogen c-myc by an internal ribosome entry site. J Biol Chem 1997, 272(51): 32061-6.
36. Yang, Q. and Sarnow, P. Location of the internal ribosome entry site in the 5′ non-coding region of the immunoglobulin heavy-chain binding protein (BiP) mRNA: evidence for specific RNA-protein interactions. Nucleic Acid Res 1997, 25(14): 2800-7.
37. Sachs, A., Sarnow, P. and Hentze, M. Starting at the beginning, middle, and end: Translation initiation in eukaryotes. Cell 1997, 89(6): 831-8.
38. Martinez-Salas, E. The impact of RNA structure on picornavirus IRES activity. Trends Microbiol 2008, 16(5): 230-7.
39. Kieft, J. Viral IRES RNA structures and ribosome interactions. Trends Biochem Sci 2008, 33(6): 274-83.
40. Martinez-Salas, E, Lopez De Quinto, S, Ramos, R, Fernandez-Miragall, O. IRES elements: features of the RNA structure contributing to their activity. Biochimie 2002, 84: 755-63.
41. Belsham, G. and Sonenberg, N. Picornavirus RNA translation: roles for cellular proteins. Trends Microbiol 2000, 8(7): 330-5.
42. Martínez-Salas, E., Ramos, R., Lafuente, E. and López de Quinto, S. Functional interactions in internal translation initiation directed by viral and cellular IRES elements. J Gen Virol 2001, 82(Pt. 5): 973-84.
43. Sugimoto, Y., Aksentijevich, I., Murray, G., Brady, R., Pastan, I. and Gottesman, M. Retroviral co-expression of a multidrug resistance gene (MDR1) and human agalactosidase A for gene therapy of Fabry disease. Hum Gene Ther 1995, 6(7): 905-15.
44. Suzuki, M., Sugimoto, Y., Tsukahara, S., Okochi, E., Gottesman, M. and Tsuruo, T. Retroviral coexpression of two different types of drug resistance genes to protect normal cells from combination chemotherapy. Clin Cancer Res 1997, 3(6): 947-54.
45. Mizuguchi, H. and Xu, Z. IRES-dependent second gene expression is significantly lower than cap-dependent first gene expression in a bicistronic vector. Mol Ther 2000, 1(4): 376-82.
46. Flasshove, M., Bardenheuer, W., Schneider, A, et. al. Type and position of promoter elements in retroviral vectors have substantial effects on the expression level of an enhanced green fluorescent protein reporter gene. J Cancer Res Clin Oncol 2000, 126(7): 391-9.
47. Oumard, A., Hennecke, M., Hauser, H. and Nourbakhsh, M. Translation of NRF mRNA is mediated by highly efficient internal ribosome entry. Mol Cell Biol 2000, 20(8): 2755-9.
48. Wong, E., Ngoi, S. and Lee, C. Improved co-expression of multiple genes in vectors containing internal ribosome entry sites (IRESes) ftorn human genes. Gene Ther 2002, 9(5): 337-44.
49. Jackson, R. and Kaminski, A. Internal initiation of translation in eukaryotes: The picornavirus paradihm and beyond. RNA 1995, 1(10): 985-1000.
50. Hennecke, M. and Kwissa, M. Composition and arrangement of genes define the strength of IRES-driven translation in bicistronic mRNAs. Nucleic Acids Res 2001, 29(16): 3327-34.
51. Borman, A., Le Mercier, P., Girard, M. and Kean, K. Comparison of picornaviral IRES-driven internal initiation of translation in cultured cells of different origins. Nucleic Acids Res 1997, 25(5): 925-32.
52. Dupuy, F., Mouly, E., Mesel-Lemoine, M. et al. Lentiviral transduction of human hematopoietic cells by HIV-1- and SIV-based vectors containing a bicistronic cassette driven by various internal promoters. J Gene Med 2005, 7(9): 1158-71.
53. Lindemann, C., Schilz, A., Emons, B. et al. Down-regulation of retroviral transgene expression during differentiation of progenitor-derived dendritic cells. Exp Hematol 2002, 30(2): 150-7.
54. Scheidemann, F., Therrien, J. and Pfützner, W. Bicistronic vectors in skin gene therapy. Arch Derm Res 2008, 300(8): 415-23.
55. Stewart, S. and Semler, B. RNA determinants of picornavirus cap-independent translation initiation. Sem Virol 1997, 8: 242-55.
56. Del Angel, R., Papavassiliou, A., Fernandez-Thomas, C., Silverman, S. and Racaniello, V. Cell proteins bind to multiple sites within the 5′ untranslated region of poliovirus RNA. Proc Natl Acad Sci U S A 1989, 86(21): 8299-303.
57. Li, M., Hantzopoulos, P., Banerjee, D., Zhao, S., Schweitzer, B., Gilbon, E. and Bertino, J. Comparison of the expression of a mutant dihydrofolate reductase under control of different internal promoters in retroviral vectors. Hum Gene Ther 1992, 3(4): 381-90.
58. Adam, M., Ramesh, N., Miller, A. and Osborne, W. Internal initiation of translation in retroviral vectors carrying picornavirus 5′ nontranslated regions. J Virol 1991, 65(9): 4985-90.
59. Arfi, A., Zisling, R., Richard, E., Batista, L., Poenaru, L., Futerman, A. and Caillaud, C. Reversion of the biochemical defects in murine embryonic Sandhoff neurons using a bicistronic lentiviral vector encoding hexosaminidase alpha and beta. J Neurochem 2006, 96(6): 1572-9.
60. Salesse, S., Lagarde, V., Ged, C., De Verneuil, H., Reiffers, J. and Mahon, F. Retroviral coexpression of IFN-alpha and IFN-gamma genes and inhibitory effects in chronic myeloid leukemia cells. J Interferon Cytokine Res 2000, 20: 577-87.
61. Moffat, M., Harmon, S., Haycock, J. and O'Malley, K. L-Dopa and dopamine-producing gene cassettes for gene therapy approaches to Parkinson's disease. Exp Neurol 1997, 144(1): 69-73.
62. Harries, M., Phillipps, N., Anderson, R., Prentice, G. and Collins, M. Comparison of bicistronic retroviral vectors containing internal ribosomal entry sites (IRES) using expression of human interleukin-12 (IL-12) as a readout. J Gene Med 2000, 2(4): 243-9.
63. Govaerts, A,, Guillaume, T., Andre, M. et al. Retroviral-mediated transfer of genes encoding interleukin-2 and interleukin-12 into fibroblasts increases host antitumor responsiveness. Cancer Gene Ther 1999, 6(5): 447-55.
64. Christensen, R., Kolvraa S, Blaese, R. and Jensen, T. Development of a skin-based metabolic sink for phenylalanine by overexpression of phenylalanine hydroxylase and GTP cyclohydrolase in primary human keratinocytes. Gene Ther 2000, 7(23): 1971-8.
65. Grez, M., Becker, S., Saulnier, S. et al. Gene therapy of chronic granulomatous disease. Bone Marrow Transplant 2000, 25(Suppl. 2): S99-104.
66. Licht, T., Haskins, M., Henthorn, P. et al. Drug selection with paclitaxel restores expression of linked IL-2 receptor gamma-chain and multidrug resistance (MDRI) transgenes in canine bone marrow. Proc Natl Acad Sci U S A 2002, 99(5): 3123-8.
67. Persons, D., Allay, E., Sawai, N. et al. Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic cells. Blood 2003, 102(2): 506-13.
68. Richard, E., Robert, E., Cario-Andre, M. et al. Hematopoietic stem cell gene therapy of murine protoporphyria by methylguanine-DNA-methyltransferase-mediated in vivo drug selection. Gene Ther 2004, 11(22): 1638-47.
69. Brenner, M. Resistance is futile. Gene Ther 1999, 6(10): 1646-7.
70. Pfützner, W. and Vogel, J. Advances in skin gene therapy. Exp Opin Invest Drugs 2000, 9: 2069-83.
71. Pfützner, W., Terunuma, A., Tock, C., et al. Topical colchicine selection of keratinocytes transduced with the multidrug resistance gene (MDRI) can sustain and enhance transgene expression in vivo. Proc Natl Acad Sci U S A 2002, 99(20): 13096-101.
72. Scheidemann, F., Loeser, M., Kromminga, A., Therrien, J., Vogel, J. and Pfützner, W. The skin as a biofactory for secretion of erythropoietin: potential of genetically modified keratinocytes and fibroblasts. Exp Dermatol 2008, 17(6): 481-8.
73. Therrien, J. Pfützner, W. and Vogel, J. An approach to achieve long-term expression in skin gene therapy. Toxicol Path 2008, 36: 104-11.
74. Takebe, N., Zhao, S. and Adhikari, D. Generation of dual resistance to 4-hydroxyperoxycyclophosphamide and methotrexate by retroviral transfer of the human aldehyde dehydrogenase class I gene and a mutated dihydrofolate reductase gene. Mol Ther 2001, 3(1): 88-96.
75. Rappa, G., Lorico, A., Hildinger, M., Fodstad, O. and Baum, C. Novel bicistronic vector expressing gamma-glutamylcysteine synthetase and the multidrug resistance protein 1 (MRP1) protects cells from MRP1-effluxed drugs and alkylating agents. Hum Gene Ther 2001, 12(14): 1785-96.
76. Oian, H., Liu, G., Can, S., Feng, J. and Ye, X. The experimental study of ovarian carcinoma vaccine modified by human B7-1 and IFN-gamma genes. Int J Gynecol Cancer 2002, 12(1): 80-5.
77. Parney, I., Petruk, K., Zhang, C., Farr-Jones, M., Sykes, D. and Chang, L. Granulocyte-macrophage colony-stimulating factor and B7-2 combination immunogene therapy in an allogeneic Hu-PBL-SCIDIbeige mouse-human glioblastoma multiforme model. Hum Gene Ther 1997, 8(9): 1073-85.
78. Warren, P., Song, W, Holle, E. et al. Combined HSV-TK/GCV and secondary lymphoid tissue chemokine gene therapy inhibits tumor growth and elicits potent antitumor CTL response in tumor-bearing mice. Anticancer Res 2002, 22(2A): 599-604.
79. Palù, G., Cavaggioni, A., Calvi, P. et al. Gene therapy of glioblastoma multiforme via combined expression of suicide and cytokine genes: a pilot study in humans. Gene Ther 1999, 6(3): 330-7.
80. Ramsubir, S., Yoshimitsu, M. and Medin, J. Ant-CD25 targeted killing of bicistronically transduced cells: a novel safety mechanism against retroviral genotoxicity. Mol Ther 2007, 15(6): 1174-81.
81. Modlich, U., Kustikova, O., Schmidt, M. et al. Leukemias following retroviral transfer of multidrug resistance 1 (MDRI) are driven by combinatorial insertional mutagenesis. Blood 2005, 105(11): 4235-46.
82. Hellen, C. AND Sarnow, P. Internal ribosome entry sites in eukaryotic mRNA molecules. Genes Dev 2001, 15(13): 1593-612.
83. Henis-Korenblit, S., Shani, G, Sines, T., Marash, L., Shobat, G. and Kimchi, A. The caspase-cleaved DAP5 protein supports internal ribosome entry site-mediated translation of death proteins. Proc Natl Acad Sci U S A 2002, 99(8): 5400-5.
84. Bonnal, S., Boutonnet, C., Prado-Lourenco, L. and. Vaguer, S. IRESdb: The Internal Ribosome Entry Site database. Nucleic Acids Res 2003, 31(1): 427-8.
85. Metz, M., Pichler, A., Kuchler, K. and Kane, S. Construction and characterization of single-transcript tricistronic retroviral vectors using two internal ribosome entry sites. Somat Cell Mol Genet 1998, 24(1): 53-69.
86. Fan, L., Owen, J. and Dickson, G. Construction and characterization of polycistronic retroviral vectors for sustained and high-level co-expression of apolipoprotein A-1 and lecithin-cholesterol acyltransferase. Atherosclerosis 1999, 147(1): 139-45.
87. De Felipe, P. and Izquierdo, M. Tricistronic and tetracistronic retroviral vectors for gene transfer. Hum Gene Ther 2000, 11(13): 1921-31.