Edelstein ML, Abedi MR, Wixon J, Edelstein RM: Gene therapy clinical trials worldwide 1989–2004-an overview. J Gene Med. 2004, 6 (6): 597-602. 10.1002/jgm.619.
Article
Google Scholar
Tomanin R, Scarpa M: Why do we need new gene therapy viral vectors? Characteristics, limitations and future perspectives of viral vector transduction. Curr Gene Ther. 2004, 4 (4): 357-372.
Article
CAS
Google Scholar
Vandendriessche TC: Clinical gene therapy – first international conference. 24–26 January 2002, Groningen, the Netherlands. IDrugs. 2002, 5 (3): 209-212.
Google Scholar
Li C, Bowles DE, van Dyke T, Samulski RJ: Adeno-associated virus vectors: potential applications for cancer gene therapy. Cancer Gene Ther. 2005
Google Scholar
Grimm D, Kay MA: From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther. 2003, 3 (4): 281-304. 10.2174/1566523034578285.
Article
CAS
Google Scholar
Nakai H, Yant SR, Storm TA, Fuess S, Meuse L, Kay MA: Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol. 2001, 75 (15): 6969-6976. 10.1128/JVI.75.15.6969-6976.2001.
Article
CAS
Google Scholar
Wang B, Li J, Xiao X: Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci USA. 2000, 97 (25): 13714-13719. 10.1073/pnas.240335297.
Article
CAS
Google Scholar
Xu A, Song L, Wang C, Wang A, Xu Q, Xiao Z, Wang S, Li M, Hao S, Li Z: [An observation of the immuno-persistence after inoculating with the domestic BRD II strain rubella vaccine among infants and young children]. Zhonghua Liu Xing Bing Xue Za Zhi. 2000, 21 (2): 117-120.
CAS
Google Scholar
Flotte TR, Solow R, Owens RA, Afione S, Zeitlin PL, Carter BJ: Gene expression from adeno-associated virus vectors in airway epithelial cells. Am J Respir Cell Mol Biol. 1992, 7 (3): 349-356.
Article
CAS
Google Scholar
Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O'Malley KL, During MJ: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet. 1994, 8 (2): 148-154. 10.1038/ng1094-148.
Article
CAS
Google Scholar
Gossen M, Bujard H: Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA. 1992, 89 (12): 5547-5551. 10.1073/pnas.89.12.5547.
Article
CAS
Google Scholar
Gossen M, Freundlieb S, Bender G, Muller G, Hillen W, Bujard H: Transcriptional activation by tetracyclines in mammalian cells. Science. 1995, 268 (5218): 1766-1769. 10.1126/science.7792603.
Article
CAS
Google Scholar
Fussenegger M, Morris RP, Fux C, Rimann M, von Stockar B, Thompson CJ, Bailey JE: Streptogramin-based gene regulation systems for mammalian cells. Nat Biotechnol. 2000, 18 (11): 1203-1208. 10.1038/81208.
Article
CAS
Google Scholar
Weber W, Fux C, Daoud-el Baba M, Keller B, Weber CC, Kramer BP, Heinzen C, Aubel D, Bailey JE, Fussenegger M: Macrolide-based transgene control in mammalian cells and mice. Nat Biotechnol. 2002, 20 (9): 901-907. 10.1038/nbt731.
Article
CAS
Google Scholar
Zhao HF, Boyd J, Jolicoeur N, Shen SH: A coumermycin/novobiocin-regulated gene expression system. Hum Gene Ther. 2003, 14 (17): 1619-1629. 10.1089/104303403322542266.
Article
CAS
Google Scholar
Rivera VM, Clackson T, Natesan S, Pollock R, Amara JF, Keenan T, Magari SR, Phillips T, Courage NL, Cerasoli F, et al: A humanized system for pharmacologic control of gene expression. Nat Med. 1996, 2 (9): 1028-1032. 10.1038/nm0996-1028.
Article
CAS
Google Scholar
Braselmann S, Graninger P, Busslinger M: A selective transcriptional induction system for mammalian cells based on Gal4-estrogen receptor fusion proteins. Proc Natl Acad Sci USA. 1993, 90 (5): 1657-1661. 10.1073/pnas.90.5.1657.
Article
CAS
Google Scholar
Beerli RR, Schopfer U, Dreier B, Barbas CF: Chemically regulated zinc finger transcription factors. J Biol Chem. 2000, 275 (42): 32617-32627. 10.1074/jbc.M005108200.
Article
CAS
Google Scholar
Chartier C, Degryse E, Gantzer M, Dieterle A, Pavirani A, Mehtali M: Efficient generation of recombinant adenovirus vectors by homologous recombination in Escherichia coli. J Virol. 1996, 70 (7): 4805-4810.
CAS
Google Scholar
Boorsma M, Nieba L, Koller D, Bachmann MF, Bailey JE, Renner WA: A temperature-regulated replicon-based DNA expression system. Nat Biotechnol. 2000, 18 (4): 429-432. 10.1038/74493.
Article
CAS
Google Scholar
Fux C, Weber W, Daoud-El Baba M, Heinzen C, Aubel D, Fussenegger M: Novel macrolide-adjustable bidirectional expression modules for coordinated expression of two different transgenes in mice. J Gene Med. 2003, 5 (12): 1067-1079. 10.1002/jgm.443.
Article
CAS
Google Scholar
Weber W, Marty RR, Link N, Ehrbar M, Keller B, Weber CC, Zisch AH, Heinzen C, Djonov V, Fussenegger M: Conditional human VEGF-mediated vascularization in chicken embryos using a novel temperature-inducible gene regulation (TIGR) system. Nucleic Acids Res. 2003, 31 (12): e69-10.1093/nar/gng069.
Article
Google Scholar
Neddermann P, Gargioli C, Muraglia E, Sambucini S, Bonelli F, De Francesco R, Cortese R: A novel, inducible, eukaryotic gene expression system based on the quorum-sensing transcription factor TraR. EMBO Rep. 2003, 4 (2): 159-165. 10.1038/sj.embor.embor734.
Article
CAS
Google Scholar
Weber W, Rimann M, Spielmann M, Keller B, Daoud-El Baba M, Aubel D, Weber CC, Fussenegger M: Gas-inducible transgene expression in mammalian cells and mice. Nat Biotechnol. 2004, 22 (11): 1440-1444. 10.1038/nbt1021.
Article
CAS
Google Scholar
Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, Bennicelli J, Dejneka NS, Pearce-Kelling SE, Maguire AM, Palczewski K, et al: Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness. Mol Ther. 2005
Google Scholar
Haberman RP, Samulski RJ, McCown TJ: Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med. 2003, 9 (8): 1076-1080. 10.1038/nm901.
Article
CAS
Google Scholar
Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, Taylor G, Walden S, Wetzel R: A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther. 1996, 7 (9): 1145-1159.
Article
CAS
Google Scholar
Chtarto A, Bender HU, Hanemann CO, Kemp T, Lehtonen E, Levivier M, Brotchi J, Velu T, Tenenbaum L: Tetracycline-inducible transgene expression mediated by a single AAV vector. Gene Ther. 2003, 10 (1): 84-94. 10.1038/sj.gt.3301838.
Article
CAS
Google Scholar
Gafni Y, Pelled G, Zilberman Y, Turgeman G, Apparailly F, Yotvat H, Galun E, Gazit Z, Jorgensen C, Gazit D: Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system. Mol Ther. 2004, 9 (4): 587-595. 10.1016/j.ymthe.2003.12.009.
Article
CAS
Google Scholar
Bohl D, Salvetti A, Moullier P, Heard JM: Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood. 1998, 92 (5): 1512-1517.
CAS
Google Scholar
Jiang L, Rampalli S, George D, Press C, Bremer EG, O'Gorman MR, Bohn MC: Tight regulation from a single tet-off rAAV vector as demonstrated by flow cytometry and quantitative, real-time PCR. Gene Ther. 2004, 11 (13): 1057-1067. 10.1038/sj.gt.3302245.
Article
CAS
Google Scholar
Rivera VM, Gao GP, Grant RL, Schnell MA, Zoltick PW, Rozamus LW, Clackson T, Wilson JM: Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood. 2005, 105 (4): 1424-1430. 10.1182/blood-2004-06-2501.
Article
CAS
Google Scholar
Wang J, Voutetakis A, Papa M, Rivera VM, Clackson T, Lodde BM, Mineshiba F, Baum BJ: Rapamycin control of transgene expression from a single AAV vector in mouse salivary glands. Gene Ther. 2005
Google Scholar
Grimm D, Kern A, Rittner K, Kleinschmidt JA: Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther. 1998, 9 (18): 2745-2760.
Article
CAS
Google Scholar
Xiao X, Li J, Samulski RJ: Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol. 1998, 72 (3): 2224-2232.
CAS
Google Scholar
Clark KR, Liu X, McGrath JP, Johnson PR: Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum Gene Ther. 1999, 10 (6): 1031-1039. 10.1089/10430349950018427.
Article
CAS
Google Scholar
Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I, Potter M, Chesnut K, Summerford C, Samulski RJ, Muzyczka N: Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 1999, 6 (6): 973-985. 10.1038/sj.gt.3300938.
Article
CAS
Google Scholar
Grimm D, Kay MA, Kleinschmidt JA: Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther. 2003, 7 (6): 839-850. 10.1016/S1525-0016(03)00095-9.
Article
CAS
Google Scholar
Weber W, Fussenegger M: Pharmacologic transgene control systems for gene therapy. J Gene Med. 2006
Google Scholar
Check E: Regulators split on gene therapy as patient shows signs of cancer. Nature. 2002, 419 (6907): 545-546. 10.1038/419545a.
Article
CAS
Google Scholar
Lehrman S: Virus treatment questioned after gene therapy death. Nature. 1999, 401 (6753): 517-518. 10.1038/43977.
Article
CAS
Google Scholar
Food and Drug Administration. [http://www.fda.gov/]
AHF Drug Information. [http://www.ashp.org/ahfs/index.cfm]
Stieger K, Le Meur G, Lasne F, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Martin L, Moullier P, et al: Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors. Mol Ther. 2006
Google Scholar
Haberman RP, McCown TJ, Samulski RJ: Inducible long-term gene expression in brain with adeno-associated virus gene transfer. Gene Ther. 1998, 5 (12): 1604-1611. 10.1038/sj.gt.3300782.
Article
CAS
Google Scholar
Markusic D, Oude-Elferink R, Das AT, Berkhout B, Seppen J: Comparison of single regulated lentiviral vectors with rtTA expression driven by an autoregulatory loop or a constitutive promoter. Nucleic Acids Res. 2005, 33 (6): e63-10.1093/nar/gni062.
Article
Google Scholar
Mitta B, Weber CC, Fussenegger M: In vivo transduction of HIV-1-derived lentiviral particles engineered for macrolide-adjustable transgene expression. J Gene Med. 2005
Google Scholar
Mitta B, Weber CC, Rimann M, Fussenegger M: Design and in vivo characterization of self-inactivating human and non-human lentiviral expression vectors engineered for streptogramin-adjustable transgene expression. Nucleic Acids Res. 2004, 32 (12): e106-10.1093/nar/gnh104.
Article
Google Scholar
Pluta K, Luce MJ, Bao L, Agha-Mohammadi S, Reiser J: Tight control of transgene expression by lentivirus vectors containing second-generation tetracycline-responsive promoters. J Gene Med. 2005, 7 (6): 803-817. 10.1002/jgm.712.
Article
CAS
Google Scholar
Vigna E, Amendola M, Benedicenti F, Simmons AD, Follenzi A, Naldini L: Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector. Mol Ther. 2005, 11 (5): 763-775. 10.1016/j.ymthe.2004.11.017.
Article
CAS
Google Scholar
Mitta B, Rimann M, Ehrengruber MU, Ehrbar M, Djonov V, Kelm J, Fussenegger M: Advanced modular self-inactivating lentiviral expression vectors for multigene interventions in mammalian cells and in vivo transduction. Nucleic Acids Res. 2002, 30 (21): e113-10.1093/nar/gnf112.
Article
Google Scholar
Schlatter S, Rimann M, Kelm J, Fussenegger M: SAMY, a novel mammalian reporter gene derived from Bacillus stearothermophilus alpha-amylase. Gene. 2002, 282 (1–2): 19-31. 10.1016/S0378-1119(01)00824-1.
Article
CAS
Google Scholar
Fux C, Fussenegger M: Toward higher order control modalities in mammalian cells-independent adjustment of two different gene activities. Biotechnol Prog. 2003, 19 (1): 109-120. 10.1021/bp0255863.
Article
CAS
Google Scholar
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, 6 (10): 1159-1169. 10.1002/jgm.601.
Article
CAS
Google Scholar
Weber W, Marty RR, Keller B, Rimann M, Kramer BP, Fussenegger M: Versatile macrolide-responsive mammalian expression vectors for multiregulated multigene metabolic engineering. Biotechnol Bioeng. 2002, 80 (6): 691-705. 10.1002/bit.10461.
Article
CAS
Google Scholar
Fussenegger M, Mazur X, Bailey JE: pTRIDENT, a novel vector family for tricistronic gene expression in mammalian cells. Biotechnol Bioeng. 1998, 57 (1): 1-10. 10.1002/(SICI)1097-0290(19980105)57:1<1::AID-BIT1>3.0.CO;2-M.
Article
CAS
Google Scholar