Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector

Molecular Therapy

Volumn 11, Issue 5, 2005, Pages 763-775

  Vigna, Elisa ^a   Amendola, Mario ^b,c   Benedicenti, Fabrizio ^b   Simmons, Andrew D ^d   Follenzi, Antonia ^a,e   Naldini, Luigi ^b,c  

^a UNIVERSITY OF TURIN   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^d CELL GENESYS INC   (United States)

^e ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

Author keywords

Gene therapy; Hemophilia B; Lentiviral vectors; Regulation of gene expression; Tet regulated system; Tissue specific promoter

Indexed keywords

BLOOD CLOTTING FACTOR 9; DOXYCYCLINE; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; TETRACYCLINE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; GENE EXPRESSION REGULATION; HEMOPHILIA B; HUMAN; HUMAN CELL; LIVER CELL; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; SCID MOUSE; TRANSCRIPTION REGULATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; CELL LINE; FACTOR IX; GENE EXPRESSION; GENETIC VECTORS; HUMANS; LENTIVIRUS; MICE; MICE, SCID; MOLECULAR SEQUENCE DATA; ORGAN SPECIFICITY; REPRODUCIBILITY OF RESULTS; TETRACYCLINE;

EID: 17644388429     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.11.017     Document Type: Article

References (43)

1. M.A. Kay J.C. Glorioso and L. Naldini Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics Nat. Med. 7 2001 33-40
2. C. Toniatti H. Bujard R. Cortese and G. Ciliberto Gene therapy progress and prospects: Transcription regulatory systems Gene Ther. 11 2004 649-657
3. U. Baron and H. Bujard Tet repressor-based system for regulated gene expression in eukaryotic cells: Principles and advances Methods Enzymol. 327 2000 401-421
4. 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
5. E. Vigna and L. Naldini Lentiviral vectors: Excellent tools for experimental gene transfer and promising candidates for gene therapy J. Gene Med. 2 2000 308-316
6. R. Mitchell C.Y. Chiang C. Berry and F. Bushman Global analysis of cellular transcription following infection with an HIV-based vector Mol. Ther. 8 2003 674-687
7. E. Vigna et al. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors Mol. Ther. 5 2002 252-261
8. 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
9. J.K. Koponen 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
10. T. Kafri H. van Praag F.H. Gage and I.M. Verma Lentiviral vectors: regulated gene expression Mol. Ther. 1 2000 516-521
11. A. Bahi F. Boyer T. Kafri and J.L. Dreyer CD81-induced behavioural changes during chronic cocaine administration: In vivo gene delivery with regulatable lentivirus Eur. J. Neurosci. 19 2004 1621-1633
12. 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
13. K.A. High Gene transfer as an approach to treating hemophilia Semin. Thromb. Hemostasis 29 2003 107-120
14. A. Follenzi L.E. Ailles S. Bakovic M. Geuna and L. Naldini Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences Nat. Genet. 25 2000 217-222
15. F. Park K. Ohashi and M.A. Kay Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver Blood 96 2000 1173-1176
16. L.V. Tsui et al. Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector Nat. Biotechnol. 20 2002 53-57
17. A. Follenzi M. Battaglia A. Lombardo A. Annoni M.G. Roncarolo and L. Naldini Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice Blood 103 2004 3700-3709
18. A. Pfeifer et al. Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging Mol. Ther. 3 2001 319-322
19. K.W. Peng et al. Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors Gene Ther. 8 2001 1456-1463
20. A. Follenzi G. Sabatino A. Lombardo C. Boccaccio and L. Naldini Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors Hum. Gene Ther. 13 2002 243-260
21. T. VandenDriessche et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo Blood 100 2002 813-822
22. D. Pan et al. Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow Mol. Ther. 6 2002 19-29
23. F. Lotti et al. Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement J. Virol. 76 2002 3996-4007
24. P. Salmon V. Kindler O. Ducrey B. Chapuis R.H. Zubler and D. Trono High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors Blood 96 2000 3392-3398
25. B. Van Maele J. De Rijck E. De Clercq and Z. Debyser Impact of the central polypurine tract on the kinetics of human immunodeficiency virus type 1 vector transduction J. Virol. 77 2003 4685-4694
26. T. Dull et al. A third-generation lentivirus vector with a conditional packaging system J. Virol. 72 1998 8463-8471
27. M. De Palma M.A. Venneri and L. Naldini In vivo targeting of tumor endothelial cells by systemic delivery of lentiviral vectors Hum. Gene Ther. 14 2003 1193-1206
28. K. Haack S.A. Cockrell H. Ma D. Israeli S.N. Ho T.J. McCown and T. Kafri Transactivator and structurally optimized inducible lentiviral vectors Mol. Ther. 10 2004 585-596
29. M.A. Srour et al. Regulation of human factor IX expression using doxycycline-inducible gene expression system Thromb. Haemostasis 90 2003 398-405
30. S. Lamartina et al. Construction of an rtTA2(s)-m2/tts(kid)-based transcription regulatory switch that displays no basal activity, good inducibility, and high responsiveness to doxycycline in mice and non-human primates Mol. Ther. 7 2003 271-280
31. U. Baron M. Gossen and H. Bujard Tetracycline-controlled transcription in eukaryotes: Novel transactivators with graded transactivation potential Nucleic Acids Res. 25 1997 2723-2729
32. D. Bohl A. Salvetti P. Moullier and J.M. Heard Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector Blood 92 1998 1512-1517
33. L. Aurisicchio et al. Regulated and prolonged expression of mIFN(alpha) in immunocompetent mice mediated by a helper-dependent adenovirus vector Gene Ther. 8 2001 1817-1825
34. K.G. Rendahl et al. Tightly regulated long-term erythropoietin expression in vivo using tet-inducible recombinant adeno-associated viral vectors Hum. Gene Ther. 13 2002 335-342
35. S. Urlinger U. Baron M. Thellmann M.T. Hasan H. Bujard and W. Hillen Exploring the sequence space for tetracycline-dependent transcriptional activators: Novel mutations yield expanded range and sensitivity Proc. Natl. Acad. Sci. USA 97 2000 7963-7968
36. C. Esslinger et al. In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8(+) T cell responses J. Clin. Invest. 111 2003 1673-1681
37. M.J. Palmowski L. Lopes Y. Ikeda M. Salio V. Cerundolo and M.K. Collins Intravenous injection of a lentiviral vector encoding NY-ESO-1 induces an effective CTL response J. Immunol. 172 2004 1582-1587
38. L. Pastore et al. Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors Hum. Gene Ther. 10 1999 1773-1781
39. A. Ehrhardt H. Xu A.M. Dillow D.A. Bellinger T.C. Nichols and M.A. Kay A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia Blood 102 2003 2403-2411
40. F. Mingozzi et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer J. Clin. Invest. 111 2003 1347-1356
41. R.H. Costa and D.R. Grayson Site-directed mutagenesis of hepatocyte nuclear factor (HNF) binding sites in the mouse transthyretin (TTR) promoter reveal synergistic interactions with its enhancer region Nucleic Acids Res. 19 1991 4139-4145
42. X. Li E.M. Eastman R.J. Schwartz and R. Draghia-Akli Synthetic muscle promoters: Activities exceeding naturally occurring regulatory sequences Nat. Biotechnol. 17 1999 241-245
43. M. De Palma E. Montini F.R. Santoni de Sio F. Benedicenti A. Gentile E. Medico and L. Naldini Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells Blood 2004