Transactivator and structurally optimized inducible lentiviral vectors

Molecular Therapy

Volumn 10, Issue 3, 2004, Pages 585-596

  Haack, Karin ^a   Cockrell, Adam S ^a   Ma, Hong ^a   Israeli, David ^b   Ho, Steffan N ^c   McCown, Thomas J ^a   Kafri, Tal ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^b GÉNÉTHON   (France)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Brain; Gene therapy; Inducible; Lentivirus vector; MyoD; Tetracycline; Transactivator

Indexed keywords

DOXYCYCLINE; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; LUCIFERASE;

ANIMAL CELL; ARTICLE; CELL SELECTION; CELL TYPE; CONTROLLED STUDY; FLUORESCENCE ACTIVATED CELL SORTER; GENE EXPRESSION; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; IMMUNOFLUORESCENCE; IN VIVO STUDY; MOUSE; NONHUMAN; NORTHERN BLOTTING; RAT; TRANSACTIVATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; WESTERN BLOTTING;

CUCUMBER MOSAIC VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS;

EID: 4444293504     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.06.109     Document Type: Article

References (37)

1. Cockrell A.S. Kafri T. HIV-1 vectors: fulfillment of expectations, further advancements, and still a way to go Curr. HIV Res. 1 2003 419-439
2. Gossen M. Bujard H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters Proc. Natl. Acad. Sci. USA 89 1992 5547-5551
3. Brown E.J. et al. A mammalian protein targeted by G1-arresting rapamycin-receptor complex Nature 369 1994 756-758
4. Wang Y. O'Malley B.W.Jr. Tsai S.Y. O'Malley B.W. A regulatory system for use in gene transfer Proc. Natl. Acad. Sci. USA 91 1994 8180-8184
5. No D. Yao T.P. Evans R.M. Ecdysone-inducible gene expression in mammalian cells and transgenic mice Proc. Natl. Acad. Sci. USA 93 1996 3346-3351
6. Hwang J.J. Li L. Anderson W.F. A conditional self-inactivating retrovirus vector that uses a tetracycline-responsive expression system J. Virol. 71 1997 7128-7131
7. Suhr S.T. Gil E.B. Senut M.C. Gage F.H. High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor Proc. Natl. Acad. Sci. USA 95 1998 7999-8004
8. Gossen M. Freundlieb S. Bender G. Müller G. Hillen W. Bujard H. Transcriptional activation by tetracyclines in mammalian cells Science 268 1995 1766-1769
9. Baron U. Gossen M. Bujard H. Tetracycline-controlled transcription in eukaryotes: novel transactivators with graded transactivation potential Nucleic Acids Res. 25 1997 2723-2739
10. Blau H.M. Rossi F.M.V. Tet B or not tet B: advances in tetracycline-inducible gene expression Proc. Natl. Acad. Sci. USA 96 1999 797-799
11. Kafri T. van Praag H. Gage F.H. Verma I.M. Lentiviral vectors: regulated gene expression Mol. Ther. 1 2000 516-521
12. Reiser J. Lai Z. Zhang X.-Y. Brady R.O. Development of multigene and regulated lentivirus vectors J. Virol. 74 2000 10589-10599
13. Xu K. Ma H. McCrown T.J. Verma I.M. Kafri T. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors Mol. Ther. 3 2001 97-104
14. Regulier E. Pereira de Almeida L. Sommer B. Aebischer P. Deglon N. 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
15. Koponen J.K. et al. 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
16. Vigna E. et al. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors Mol. Ther. 5 2002 252-261
17. Zufferey R. Donello J.E. Trono D. Hope T.J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors J. Virol. 73 1999 2886-2892
18. Urlinger S. Helbl V. Guthmann J. Pook E. Grimm S. Hillen W. The p65 domain from NF-κB is an efficient human activator in the tetracycline-regulatable gene expression system Gene 247 2000 103-110
19. Kitzmann M. et al. cdk1- and cdk2-mediated phosphorylation of MyoD Ser200 in growing C2 myoblasts: role in modulating MyoD half-life and myogenic activity Mol. Cell. Biol. 19 1999 3167-3176
20. Hoffmann A. Villalba M. Journot L. Spengler D. A novel tetracycline-dependent expression vector with low basal expression and potent regulatory properties in various mammalian cell lines Nucleic Acids Res. 25 1997 1078-1079
21. Leuchtenberger S. Perz A. Gatz C. Bartsch J.W. Conditional cell ablation by stringent tetracycline-dependent regulation of barnase in mammalian cells Nucleic Acids Res. 29 2001 E76
22. Schmeisser F. Donohue M. Weir J.P. Tetracycline-regulated gene expression in replication-incompetent herpes simplex virus vectors Hum. Gene Ther. 13 2002 2113-2124
23. Go W.Y. Ho S.N. Optimization and direct comparison of the dimerizer and reverse tet transcriptional control systems J. Gene Med. 4 2002 258-270
24. Fender P. et al. Controlled transgene expression by E1-E4-defective adenovirus vectors harbouring a "tet-on" switch system J. Gene Med. 4 2002 668-675
25. Favre D. et al. Lack of an immune response against the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated transgene expression in nonhuman primates after intramuscular injection of recombinant adeno-associated virus J. Virol. 76 2002 11605-11611
26. Latta-Mahieu M. et al. Gene transfer of a chimeric trans-activator is immunogenic and results in short-lived transgene expression Hum. Gene Ther. 13 2002 1611-1620
27. Chittum H.S. et al. Rabbit beta-globin is extended beyond its UGA stop codon by multiple suppressions and translational reading gaps Biochemistry 37 1998 10866-10870
28. Steneberg P. Samakovlis C. A novel stop codon readthrough mechanism produces functional Headcase protein in Drosophila trachea EMBO Rep. 2 2001 593-597
29. Beier H. Grimm M. Misreading of termination codons in eukaryotes by natural nonsense suppressor tRNAs Nucleic Acids Res. 29 2001 4767-4782
30. Namy O. Hatin I. Rousset J.P. Impact of the six nucleotides downstream of the stop codon on translation termination EMBO Rep 2 2001 787-793
31. Cassan M. Rousset J.P. UAG readthrough in mammalian cells: effect of upstream and downstream stop codon contexts reveal different signals BMC Mol. Biol. 2 2001 3-10
32. Sogaard T.M. Jakobsen C.G. Justesen J. A sensitive assay of translational fidelity (readthrough and termination) in eukaryotic cells Biochemistry (Moscow) 64 1999 1408-1417
33. Miyoshi H. Blomer U. Takahashi M. Gage F.H. Verma I.M. Development of a self-inactivating lentivirus vector J. Virol. 72 1998 8150-8157
34. Naldini L. et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector Science 272 1996 263-267
35. Kafri T. van Praag H. Ouyang L. Gage F.H. Verma I.M. A packaging cell line for lentivirus vectors J. Virol. 73 1999 576-584
36. Gregory C.W. Johnson R.T.Jr. Mohler J.L. French F.S. Wilson E.M. Androgen receptor stabilization in recurrent prostate cancer is associated with hypersensitivity to low androgen Cancer Res. 61 2001 2892--2898
37. McCown T.J. Xiao X. Li J. Breese G.R. Samulski R.J. Differential and persistent expression patterns of CNV gene transfer by an adeno-associated virus (AAV) vector Brain Res. 713 1996 99-107