Approaches for trigger-inducible viral transgene regulation in gene-based tissue engineering

Current Opinion in Biotechnology

Volumn 15, Issue 5, 2004, Pages 383-391

  Weber, Wilfried ^a   Fussenegger, Martin ^a  

^a ETH ZURICH   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBIOTIC AGENT; MACROLIDE; MIFEPRISTONE; RAPAMYCIN; RETROVIRUS VECTOR; STREPTOGRAMIN DERIVATIVE; TAMOXIFEN; TETRACYCLINE;

BACTERIOPHAGE; COMPLEX FORMATION; DNA BINDING; DRUG DELIVERY SYSTEM; DRUG DESIGN; EUKARYOTE; GENE ACTIVATION; GENE CONTROL; GENE EXPRESSION; GENE MUTATION; GENE TARGETING; GENE TRANSFER; GENE VECTOR; GENETIC TRANSCRIPTION; MAMMAL CELL; MOLECULAR INTERACTION; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROMOTER REGION; REVIEW; SIGNAL TRANSDUCTION; TANDEM REPEAT; TECHNIQUE; TISSUE ENGINEERING; TRANSACTIVATION; TRANSGENE; TRANSLATION INITIATION; VIRUS GENE; VIRUS INDUCTION;

ANIMALS; GENE EXPRESSION REGULATION; GENE THERAPY; GENES, VIRAL; GENETIC VECTORS; HUMANS; RESEARCH DESIGN; TISSUE ENGINEERING; TRANSGENES;

EUKARYOTA; MAMMALIA; UNIDENTIFIED RETROVIRUS;

EID: 4644254892     PISSN: 09581669     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.copbio.2004.07.003     Document Type: Review

References (58)

1. M. Fussenegger The impact of mammalian gene regulation concepts on functional genomic research, metabolic engineering, and advanced gene therapies Biotechnol Prog 17 2001 1 51
2. M. Gossen, and H. Bujard Tight control of gene expression in mammalian cells by tetracycline-responsive promoters Proc Natl Acad Sci USA 89 1992 5547 5551
3. S.Y. Corbel, and F.M. Rossi Latest developments and in vivo use of the Tet system: ex vivo and in vivo delivery of tetracycline-regulated genes Curr Opin Biotechnol 13 2002 448 452
4. M. Fussenegger, R.P. Morris, C. Fux, M. Rimann, B. von Stockar, C.J. Thompson, and J.E. Bailey Streptogramin-based gene regulation systems for mammalian cells Nat Biotechnol 18 2000 1203 1208
5. V.M. Rivera, T. Clackson, S. Natesan, R. Pollock, J.F. Amara, T. Keenan, S.R. Magari, T. Phillips, N.L. Courage, and F. Cerasoli Jr. A humanized system for pharmacologic control of gene expression Nat Med 2 1996 1028 1032
6. T.D. Littlewood, D.C. Hancock, P.S. Danielian, M.G. Parker, and G.I. Evan A modified oestrogen receptor ligand-binding domain as an improved switch for the regulation of heterologous proteins Nucleic Acids Res 23 1995 1686 1690
7. Y. Wang, B.W. O'Malley Jr., S.Y. Tsai, and B.W. O'Malley A regulatory system for use in gene transfer Proc Natl Acad Sci USA 91 1994 8180 8184
8. M. Gossen, S. Freundlieb, G. Bender, G. Muller, W. Hillen, and H. Bujard Transcriptional activation by tetracyclines in mammalian cells Science 268 1995 1766 1769
9. J.K. Koponen, H. Kankkonen, J. Kannasto, T. Wirth, W. Hillen, H. Bujard, and S. Yla-Herttuala Doxycycline-regulated lentiviral vector system with a novel reverse transactivator rtTA2S-M2 shows a tight control of gene expression in vitro and in vivo Gene Ther 10 2003 459 466
10. W. Weber, B.P. Kramer, C. Fux, B. Keller, and M. Fussenegger Novel promoter/transactivator configurations for macrolide- and streptogramin- responsive transgene expression in mammalian cells J Gene Med 4 2002 676 686
11. S. Tascou, T.K. Sorensen, V. Glenat, M. Wang, M.M. Lakich, R. Darteil, E. Vigne, and V. Thuillier Stringent rosiglitazone-dependent gene switch in muscle cells without effect on myogenic differentiation Mol Ther 9 2004 637 649
12. F. Yao, T. Svensjo, T. Winkler, M. Lu, C. Eriksson, and E. Eriksson Tetracycline repressor, tetR, rather than the tetR-mammalian cell transcription factor fusion derivatives, regulates inducible gene expression in mammalian cells Hum Gene Ther 9 1998 1939 1950
13. W. Weber, C. Fux, M. Daoud-el Baba, B. Keller, C.C. Weber, B.P. Kramer, C. Heinzen, D. Aubel, J.E. Bailey, and M. Fussenegger Macrolide-based transgene control in mammalian cells and mice Nat Biotechnol 20 2002 901 907
14. P. Neddermann, C. Gargioli, E. Muraglia, S. Sambucini, F. Bonelli, R. De Francesco, and R. Cortese A novel, inducible, eukaryotic gene expression system based on the quorum-sensing transcription factor TraR EMBO Rep 4 2003 159 165
15. W. Weber, R. Schoenmakers, M. Spielmann, M.D. El-Baba, M. Folcher, B. Keller, C.C. Weber, N. Link, P. van de Wetering, and C. Heinzen Streptomyces-derived quorum-sensing systems engineered for adjustable transgene expression in mammalian cells and mice Nucleic Acids Res 31 2003 e71
16. B.P. Kramer, A. Usseglio Viretta, M. Daoud-el Baba, D. Aubel, W. Weber, and M. Fussenegger Engineering of epigenetic transgene control in mammalian cells Nat Biotechnol 22 2004 867 870 The interconnection of macrolide- and streptogramin-dependent repressors, which control each other's expression, resulted in a mammalian epigenetic circuitry able to switch between two stable transgene expression states following transient administration of two alternate drugs. Engineered Chinese hamster ovary cells showed reversible toggle-switch-specific transgene expression in culture as well as following encapsulation and implantation into mice.
17. W. Weber, and M. Fussenegger Artificial mammalian gene regulation networks-novel approaches for gene therapy and bioengineering J Biotechnol 98 2002 161 187
18. Fux C, Langer D, Fussenegger M: Dual-regulated myoD- and msx1-based interventions in C2C12-derived cells enable precise myogenic/osteogenic/ adipogenic lineage control. J Gene Med 2004, in press.
19. C. Fux, B. Mitta, B.P. Kramer, and M. Fussenegger Dual-regulated expression of C/EBP-α and BMP-2 enables differential differentiation of C2C12 cells into adipocytes and osteoblasts Nucleic Acids Res 32 2004 e1
20. Kramer BP, Fischer C, Fussenegger M: BioLogic gates enable logical transcription control iin mammalian cells. Biotechnol Bioeng 2004, in press.
21. B.P. Kramer, W. Weber, and M. Fussenegger Artificial regulatory networks and cascades for discrete multilevel transgene control in mammalian cells Biotechnol Bioeng 83 2003 810 820
22. W.W. Gibbs Synthetic life Sci Am 290 2004 74 81
23. N.A. Kootstra, and I.M. Verma Gene therapy with viral vectors Annu Rev Pharmacol Toxicol 43 2003 413 439
24. J.M. Kelm, B.P. Kramer, V. Gonzalez-Nicolini, B. Ley, and M. Fussenegger Synergies of microtissue design, viral transduction and adjustable transgene expression for regenerative medicine Biotechnol Appl Biochem 39 2004 3 16
25. M. Cavazzana-Calvo, A. Thrasher, and F. Mavilio The future of gene therapy Nature 427 2004 779 781
26. H. Yoshiji, J. Yoshii, Y. Ikenaka, R. Noguchi, K. Yanase, H. Tsujinoue, H. Imazu, and H. Fukui Suppression of the renin-angiotensin system attenuates vascular endothelial growth factor-mediated tumor development and angiogenesis in murine hepatocellular carcinoma cells Int J Oncol 20 2002 1227 1231
27. N. Sato, K. Matsuda, C. Sakuma, D.N. Foster, R.W. Oppenheim, and H. Yaginuma Regulated gene expression in the chicken embryo by using replication-competent retroviral vectors J Virol 76 2002 1980 1985
28. J. Unsinger, A. Kroger, H. Hauser, and D. Wirth Retroviral vectors for the transduction of autoregulated, bidirectional expression cassettes Mol Ther 4 2001 484 489
29. M. Crittenden, M. Gough, J. Chester, T. Kottke, J. Thompson, A. Ruchatz, T. Clackson, F.L. Cosset, H. Chong, and R.M. Diaz Pharmacologically regulated production of targeted retrovirus from T cells for systemic antitumor gene therapy Cancer Res 63 2003 3173 3180 T cells were engineered for constitutive expression of a chimeric receptor specific for tumor-associated carcinoembryonic antigen (CEA) and rapamycin-inducible provirus production. The retroviral provirus had its 5′LTR replaced by the rapamycin-inducible promoter, encoded a Herpes simplex virus thymidine kinase gene and harboured a CEA-specific enhancer in its 3′LTR. Rapamycin-inducible provirus transcription following T cell-tumor cell interaction resulted in retroviral particles transcriptionally targeted to CEA-expressing tumor cells. Mice bearing metastatic tumors that received engineered T cells showed a significant increase in survival when treated with rapamycin.
30. Y. Hanazono, T. Nagashima, M. Takatoku, H. Shibata, N. Ageyama, T. Asano, Y. Ueda, C.E. Dunbar, A. Kume, and K. Terao In vivo selective expansion of gene-modified hematopoietic cells in a nonhuman primate model Gene Ther 9 2002 1055 1064
31. A. Kume, M. Koremoto, R. Xu, T. Okada, H. Mizukami, Y. Hanazono, M. Hasegawa, and K. Ozawa In vivo expansion of transduced murine hematopoietic cells with a selective amplifier gene J Gene Med 5 2003 175 181
32. D.P. Silver, and D.M. Livingston Self-excising retroviral vectors encoding the Cre recombinase overcome Cre-mediated cellular toxicity Mol Cell 8 2001 233 243
33. B. Mitta, M. Rimann, M.U. Ehrengruber, M. Ehrbar, V. Djonov, J. Kelm, and M. Fussenegger Advanced modular self-inactivating lentiviral expression vectors for multigene interventions in mammalian cells and in vivo transduction Nucleic Acids Res 30 2002 e113
34. E. Vigna, S. Cavalieri, L. Ailles, M. Geuna, R. Loew, H. Bujard, and L. Naldini Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors Mol Ther 5 2002 252 261
35. G. Tiscornia, O. Singer, M. Ikawa, and I.M. Verma A general method for gene knockdown in mice by using lentiviral vectors expressing small interfering RNA Proc Natl Acad Sci USA 100 2003 1844 1848
36. A. Pfeifer, E.P. Brandon, N. Kootstra, F.H. Gage, and I.M. Verma Delivery of the Cre recombinase by a self-deleting lentiviral vector: efficient gene targeting in vivo Proc Natl Acad Sci USA 98 2001 11450 11455
37. M. Wiznerowicz, and D. Trono Conditional suppression of cellular genes: lentivirus vector-mediated drug-inducible RNA interference J Virol 77 2003 8957 8961 First report on the virus-based tetracycline-responsive expression of siRNAs specifically silencing ectopic green fluorescent protein or endogenous p53 expression.
38. E. Regulier, L. Pereira de Almeida, B. Sommer, P. Aebischer, and N. Deglon Dose-dependent neuroprotective effect of ciliary neurotrophic factor delivered via tetracycline-regulated lentiviral vectors in the quinolinic acid rat model of Huntington's disease Hum Gene Ther 13 2002 1981 1990
39. E. Regulier, Y. Trottier, V. Perrin, P. Aebischer, and N. Deglon Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum Hum Mol Genet 12 2003 2827 2836 Huntington's disease phenotypes were reversibly triggered in rats following tetracycline-responsive expression of mutated huntingtin from transgenic lentiviral particles injected into the rat striatum.
40. R. Vogel, L. Amar, A.D. Thi, P. Saillour, and J. Mallet A single lentivirus vector mediates doxycycline-regulated expression of transgenes in the brain Hum Gene Ther 15 2004 157 165
41. J. Johansen, C. Rosenblad, K. Andsberg, A. Moller, C. Lundberg, A. Bjorlund, and T.E. Johansen Evaluation of Tet-on system to avoid transgene down-regulation in ex vivo gene transfer to the CNS Gene Ther 9 2002 1291 1301
42. B. Mitta, C.C. Weber, M. Rimann, and M. Fussenegger Design and in vivo charakterization of self-inactivating human and non-human lentiviral expression vectors engineered for streptogramin-adjustable transgene expression Nucleic Acids Res 32 2004 e106
43. O. Sirin, and F. Park Regulating gene expression using self-inactivating lentiviral vectors containing the mifepristone-inducible system Gene 323 2003 67 77
44. R.W. Carthew Gene silencing by double-stranded RNA Curr Opin Cell Biol 13 2001 244 248
45. H. Xia, Q. Mao, H.L. Paulson, and B.L. Davidson siRNA-mediated gene silencing in vitro and in vivo Nat Biotechnol 20 2002 1006 1010
46. V. Salucci, A. Scarito, L. Aurisicchio, S. Lamartina, G. Nicolaus, S. Giampaoli, O. Gonzalez-Paz, C. Toniatti, H. Bujard, and W. Hillen Tight control of gene expression by a helper-dependent adenovirus vector carrying the rtTA2(s)-M2 tetracycline transactivator and repressor system Gene Ther 9 2002 1415 1421
47. D. Zerby, K. Sakhuja, P.S. Reddy, H. Zimmerman, D. Kayda, S. Ganesh, S. Pattison, T. Brann, M.J. Kadan, and M. Kaleko In vivo ligand-inducible regulation of gene expression in a gutless adenoviral vector system Hum Gene Ther 14 2003 749 761
48. K. Takahashi, Y. Saishin, R.L. Silva, Y. Oshima, S. Oshima, M. Melia, B. Paszkiet, D. Zerby, M.J. Kadan, and G. Liau Intraocular expression of endostatin reduces VEGF-induced retinal vascular permeability, neovascularization, and retinal detachment FASEB J 17 2003 896 898
49. A. Chtarto, H.U. Bender, C.O. Hanemann, T. Kemp, E. Lehtonen, M. Levivier, J. Brotchi, T. Velu, and L. Tenenbaum Tetracycline-inducible transgene expression mediated by a single AAV vector Gene Ther 10 2003 84 94
50. A. Auricchio, V.M. Rivera, T. Clackson, E.E. O'Connor, A.M. Maguire, M.J. Tolentino, J. Bennett, and J.M. Wilson Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye Mol Ther 6 2002 238 242 AAV vectors engineered for rapamycin-inducible erythropoietin production were administered to the intravitreal or subretinal space of nude rats or the subretinal space of non-human primates. Systemic rapamycin treatment resulted in reversible and dose-dependent erythropoietin expression over several weeks.
51. R.P. Haberman, and T.J. McCown Regulation of gene expression in adeno-associated virus vectors in the brain Methods 28 2002 219 226
52. S. Folliot, D. Briot, H. Conrath, N. Provost, Y. Cherel, P. Moullier, and F. Rolling Sustained tetracycline-regulated transgene expression in vivo in rat retinal ganglion cells using a single type 2 adeno-associated viral vector J Gene Med 5 2003 493 501
53. J. Johnston, J. Tazelaar, V.M. Rivera, T. Clackson, G.P. Gao, and J.M. Wilson Regulated expression of erythropoietin from an AAV vector safely improves the anemia of β-thalassemia in a mouse model Mol Ther 7 2003 493 497
54. F. Apparailly, V. Millet, D. Noel, C. Jacquet, J. Sany, and C. Jorgensen Tetracycline-inducible interleukin-10 gene transfer mediated by an adeno-associated virus: application to experimental arthritis Hum Gene Ther 13 2002 1179 1188
55. Y. Gafni, G. Pelled, Y. Zilberman, G. Turgeman, F. Apparailly, H. Yotvat, E. Galun, Z. Gazit, C. Jorgensen, and D. Gazit Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system Mol Ther 9 2004 587 595
56. F. Schmeisser, M. Donohue, and J.P. Weir Tetracycline-regulated gene expression in replication-incompetent herpes simplex virus vectors Hum Gene Ther 13 2002 2113 2124
57. S. Wang, J. Petravicz, and X.O. Breakefield Single HSV-amplicon vector mediates drug-induced gene expression via dimerizer system Mol Ther 7 2003 790 800
58. S. Hacein-Bey-Abina, C. Von Kalle, M. Schmidt, M.P. McCormack, N. Wulffraat, P. Leboulch, A. Lim, C.S. Osborne, R. Pawliuk, and E. Morillon LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302 2003 415 419