An internal polyadenylation signal substantially increases expression levels of lentivirus-delivered transgenes but has the potential to reduce viral titer in a promoter-dependent manner

Human Gene Therapy

Volumn 19, Issue 8, 2008, Pages 840-850

  Hager, Stefanie ^a   Frame, Fiona M ^a   Collins, Anne T ^a   Burns, Julie E ^a   Maitland, Norman J ^a  

^a UNIVERSITY OF YORK   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BETA ACTIN; ELONGATION FACTOR 1ALPHA; GENOMIC RNA; LENTIVIRUS VECTOR; MESSENGER RNA; PROBASIN; PROSTATE SPECIFIC ANTIGEN;

ARTICLE; CELL STRAIN HEK293; CELL STRAIN LNCAP; CONTROLLED STUDY; CYTOMEGALOVIRUS; ENZYME LINKED IMMUNOSORBENT ASSAY; EPITHELIUM CELL; FLOW CYTOMETRY; GENE CASSETTE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE SEQUENCE; GENE TARGETING; GENETIC CODE; HUMAN; HUMAN CELL; LENTIVIRINAE; MORANGE GENE; NONHUMAN; POLYADENYLATION; PROMOTER REGION; PROSTATE CANCER; SIGNAL TRANSDUCTION; SIMIAN VIRUS 40; TISSUE SPECIFICITY; TRANSGENE; VIRUS TITRATION;

CELL LINE, TUMOR; GENE EXPRESSION; GENE THERAPY; GENES, REPORTER; GENETIC VECTORS; GENOME, VIRAL; HUMANS; LENTIVIRUS; MALE; POLYADENYLATION; PROMOTER REGIONS (GENETICS); PROSTATIC NEOPLASMS; RNA, MESSENGER; TRANSDUCTION, GENETIC; TRANSGENES;

CYTOMEGALOVIRUS; LENTIVIRUS; SIMIAN VIRUS 40;

EID: 50649087752     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2007.165     Document Type: Article

References (49)

1. Armentano, D., Yu, S.F., Kantoff, P.W., von Ruden, T., Anderson, W.F., and Gilboa, E. (1987). Effect of internal viral sequences on the utility of retroviral vectors. J. Virol. 61, 1647-1650.
2. Bandyopadhyay, P.K., and Temin, H.M. (1984). Expression of complete chicken thymidine kinase gene inserted in a retrovirus vector. Mol. Cell. Biol. 4, 749-754.
3. Blo, M., Bogenberger, J.M., Swift, S.E., Micklem, D.R., and Lorens, J.B. (2007). Expanding the spectrum of genetic elements transferable by retroviral vectors. DNA Cell Biol. 26, 773-779.
4. Blo, M., Micklem, D.R., and Lorens, J.B. (2008). Enhanced gene expression from retroviral vectors. BMC Biotechnol. 8, 19-24.
5. Buchschacher, G.L., Jr., and Wong-Staal, F. (2000). Development of lentiviral vectors for gene therapy for human diseases. Blood 95, 2499-2504.
6. Bukrinsky, M.I., Haggerty, S., Dempsey, M.P., Sharova, N., Adzhubel, A., Spitz, L., Lewis, P., Goldfarb, D., Emerman, M., and Stevenson, M. (1993). A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365, 666-669.
7. Carlotti, F., Bazuine, M., Kekarainen, T., Seppen, J., Pognonec, P., Maassen, J.A., and Hoeben, R.C. (2004). Lentiviral vectors efficiently transduce quiescent mature 3T3-L1 adipocytes. Mol. Ther. 9, 209-217.
8. Chapel-Fernandes, S., Jordier, F., Lauro, F., Maitland, N., Chiaroni, J., de Micco, P., Mannoni, P., and Bagnis, C. (2006). Use of the PSA enhancer core element to modulate the expression of prostate- and non-prostate-specific basal promoters in a lentiviral vector context. Cancer Gene Ther. 13, 919-929.
9. Choi, T., Huang, M., Gorman, C., and Jaenisch, R. (1991). A generic intron increases gene expression in transgenic mice. Mol. Cell. Biol. 11, 3070-3074.
10. Danckwardt, S., Hartmann, K., Gehring, N.H., Hentze, M.W., and Kulozik, A.E. (2006). 3′ End processing of the prothrombin mRNA in thrombophilia. Acta Haematol. 115, 192-197.
11. Ellis, J. (2005). Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum. Gene Ther. 16, 1241-1246.
12. Fehse, B., Kustikova, O.S., Bubenheim, M., and Baum, C. (2004). Pois(s)on: It's a question of dose. Gene Ther. 11, 879-881.
13. Fink, M., Flekna, G., Ludwig, A., Heimbucher, T., and Czerny, T. (2006). Improved translation efficiency of injected mRNA during early embryonic development. Dev. Dyn. 235, 3370-3378.
14. Gerolami, R., Uch, R., Faivre, J., Garcia, S., Hardwigsen, J., Cardoso, J., Mathieu, S., Bagnis, C., Brechot, C., and Mannoni, P. (2004). Herpes simplex virus thymidine kinase-mediated suicide gene therapy for hepatocellular carcinoma using HIV-1-derived lentiviral vectors. J. Hepatol. 40, 291-297.
15. Hlavaty, J., Stracke, A., Klein, D., Salmons, B., Gunzburg, W.H., and Renner, M. (2004). Multiple modifications allow high-titer production of retroviral vectors carrying heterologous regulatory elements. J. Virol. 78, 1384-1392.
16. Hlavaty, J., Schittmayer, M., Stracke, A., Jandl, G., Knapp, E., Felber, B.K., Salmons, B., Gunzburg, W.H., and Renner, M. (2005). Effect of posttranscriptional regulatory elements on transgene expression and virus production in the context of retrovirus vectors. Virology 341, 1-11.
17. Iwakuma, T., Cui, Y., and Chang, L.J. (1999). Self-inactivating lentiviral vectors with U3 and U5 modifications. Virology 261, 120-132.
18. Jackson, R.J., and Standart, N. (1990). Do the poly(A) tail and 3′ untranslated region control mRNA translation? Cell 62, 15-24.
19. Klein, R., Ruttkowski, B., Knapp, E., Salmons, B., Gunzburg, W.H., and Hohenadl, C. (2006). WPRE-mediated enhancement of gene expression is promoter and cell line specific. Gene 372, 153-161.
20. Kraaij, R., van der Weel, L., de Ridder, C.M., van der Korput, H.A., Zweistra, J.L., van Rijswijk, A.L., Bangma, C.H., and Trapman, J. (2007). A small chimeric promoter for high prostate-specific transgene expression from adenoviral vectors. Prostate 67, 829-839.
21. Kraunus, J., Schaumann, D.H., Meyer, J., Modlich, U., Fehse, B., Brandenburg, G., von Laer, D., Klump, H., Schambach, A., Bohne, J., and Baum, C. (2004). Self-inactivating retroviral vectors with improved RNA processing. Gene Ther. 11, 1568-1578.
22. 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.
23. Lois, C., Hong, E.J., Pease, S., Brown, E.J., and Baltimore, D. (2002). Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors. Science 295, 868-872.
24. Maitland, N.J., Macintosh, C.A., Hall, J., Sharrard, M., Quinn, G., and Lang, S. (2001). In vitro models to study cellular differentiation and function in human prostate cancers. Radiat. Res. 155, 133-142.
25. Maxwell, I.H., Brown, J.L., and Maxwell, F. (1991). Inefficiency of expression of luciferase reporter from transfected murine leukaemia proviral DNA may be partially overcome by providing a strong polyadenylation signal. J. Gen. Virol. 72, 1721-1724.
26. Miller, A.D., Jolly, D.J., Friedmann, T., and Verma, I.M. (1983). A transmissible retrovirus expressing human hypoxanthine phosphoribosyltransferase (HPRT): Gene transfer into cells obtained from humans deficient in HPRT. Proc. Natl. Acad. Sci. U.S.A. 80, 4709-4713.
27. Mitta, B., Rimann, M., and Fussenegger, M. (2005). Detailed design and comparative analysis of protocols for optimized production of high-performance HIV-1-derived lentiviral particles. Metab. Eng. 7, 426-436.
28. Moreau-Gaudry, F., Xia, P., Jiang, G., Perelman, N.P., Bauer, G., Ellis, J., Surinya, K.H., Mavilio, F., Shen, C.K., and Malik, P. (2001). High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors. Blood 98, 2664-2672.
29. Naldini, L., Blomer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F.H., Verma, I.M., and Trono, D. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267.
30. Narita, M., Takanaga, K., Yoshida, Y., Kadomatsu, K., Muramatsu, T., Matsubara, S., Hamada, H., Goto, S., Saisho, H., Sakiyama, S., and Tagawa, M. (2000). Polyadenylation signal facilitates the expression of foreign gene that is driven by an internal promoter located in the reverse orientation to long terminal repeat of retrovirus. Anticancer Res. 20, 279-282.
31. Nettelbeck, D.M., Jerome, V., and Muller, R. (1998). A strategy for enhancing the transcriptional activity of weak cell type-specific promoters. Gene Ther. 5, 1656-1664.
32. Piacibello, W., Bruno, S., Sanavio, F., Droetto, S., Gunetti, M., Ailles, L., Santoni de Sio, F., Viale, A., Gammaitoni, L., Lombardo, A., Naldini, L., and Aglietta, M. (2002). Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Blood 100, 4391-4400.
33. Ramezani, A., Hawley, T.S., and Hawley, R.G. (2000). Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol. Ther. 2, 458-469.
34. Salmon, P., Kindler, V., Ducrey, O., Chapuis, B., Zubler, R.H., and Trono, D. (2000). High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 96, 3392-3398.
35. Salmons, B., and Gunzburg, W.H. (1993). Targeting of retroviral vectors for gene therapy. Hum. Gene Ther. 4, 129-141.
36. Schambach, A., Wodrich, H., Hildinger, M., Bohne, J., Krausslich, H.G., and Baum, C. (2000). Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol. Ther. 2, 435-445.
37. Schambach, A., Galla, M., Maetzig, T., Loew, R., and Baum, C. (2007). Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol. Ther. 15, 1167-1173.
38. Shaner, N.C., Campbell, R.E., Steinbach, P.A., Giepmans, B.N., Palmer, A.E., and Tsien, R.Y. (2004). Improved monomeric red, orange and yellow fluorescent proteins derived from Discosoma sp. red fluorescent protein. Nat. Biotechnol. 22, 1567-1572.
39. Shimotohno, K., and Temin, H.M. (1981). Formation of infectious progeny virus after insertion of herpes simplex thymidine kinase gene into DNA of an avian retrovirus. Cell 26, 67-77.
40. Suter, D.M., Cartier, L., Bettiol, E., Tirefort, D., Jaconi, M.E., Dubois-Dauphin, M., and Krause, K.H. (2006). Rapid generation of stable transgenic embryonic stem cell lines using modular lentivectors. Stem Cells 24, 615-623.
41. Trobridge, G., and Russell, D.W. (2004). Cell cycle requirements for transduction by foamy virus vectors compared with those of oncovirus and lentivirus vectors. J. Virol. 78, 2327-2335.
42. van den Brandt, J., Wang, D., Kwon, S.H., Heinkelein, M., and Reichardt, H.M. (2004). Lentivirally generated eGFP-transgenic rats allow efficient cell tracking in vivo. Genesis 39, 94-99.
43. Werner, M., Kraunus, J., Baum, C., and Brocker, T. (2004). B-cell-specific transgene expression using a self-inactivating retroviral vector with human CD19 promoter and viral post-transcriptional regulatory element. Gene Ther. 11, 992-1000.
44. Xia, X., Zhang, Y., Zieth, C.R., and Zhang, S.C. (2007). Transgenes delivered by lentiviral vector are suppressed in human embryonic stem cells in a promoter-dependent manner. Stem Cells Dev. 16, 167-176.
45. Yee, J.K., Moores, J.C., Jolly, D.J., Wolff, J.A., Respess, J.G., and Friedmann, T. (1987). Gene expression from transcriptionally disabled retroviral vectors. Proc. Natl. Acad. Sci. U.S.A. 84, 5197-5201.
46. Yu, D., Scott, C., Jia, W.W., de Benedetti, A., Williams, B.J., Fazli, L., Wen, Y., Gleave, M., Nelson, C., and Rennie, P.S. (2006). Targeting and killing of prostate cancer cells using lentiviral constructs containing a sequence recognized by translation factor eIF4E and a prostate-specific promoter. Cancer Gene Ther. 13, 32-43.
47. Zhang, F., Thornhill, S.I., Howe, S.J., Ulaganathan, M., Schambach, A., Sinclair, J., Kinnon, C., Gaspar, H.B., Antoniou, M., and Thrasher, A.J. (2007). Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 110, 1448-1457.
48. Zhao, J., Hyman, L., and Moore, C. (1999). Formation of mRNA 3′ ends in eukaryotes: Mechanism, regulation, and interrelationships with other steps in mRNA synthesis. Microbiol. Mol. Biol. Rev. 63, 405-445.
49. 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.