Regulated expression of adenoviral vectors-based gene therapies: Therapeutic expression of toxins and immune-modulators

Methods in Molecular Biology

Volumn 434, Issue , 2008, Pages 239-266

  Curtin, James F ^a   Candolfi, Marianela ^a   Puntel, Mariana ^a   Xiong, Weidong ^a   Muhammad, A K M ^a   Kroeger, Kurt ^a   Mondkar, Sonali ^a   Liu, Chunyan ^a   Bondale, Niyati ^a   Lowenstein, Pedro R ^a   Castro, Maria G ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Adenoviral vectors; Doxycycline; Gene therapy; Glioma; Inducible; Pseudomonas exotoxin A (PE); Regulatable; TetON; Tetracycline

Indexed keywords

ADENOVIRIDAE; PSEUDOMONAS EXOTOXIN;

EID: 84934442453     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-60327-248-3_15     Document Type: Article

References (25)

1. Wurm, F. M., Gwinn, K. A., and Kingston, R. E. (1986) Inducible overproduction of the mouse c-myc protein in mammalian cells, Proc Natl Acad Sci USA 83, 5414-8.
2. Rivera, V. M., Clackson, T., Natesan, S., Pollock, R., Amara, J. F., Keenan, T., Magari, S. R., Phillips, T., Courage, N. L., Cerasoli, F., Jr., Holt, D. A., and Gilman, M. (1996) A humanized system for pharmacologie control of gene expression, Nat Med 2, 1028-32.
3. Molin, M., Shoshan, M. C., Ohman-Forslund, K., Linder, S., and Akusjarvi, G. (1998) Two novel adenovirus vector systems permitting regulated protein expression in gene transfer experiments, J Virol 72, 8358-61.
4. Goverdhana, S., Puntel, M., Xiong, W., Zirger, J. M., Barcia, C., Curtin, J. F., Soffer, E. B., Mondkar, S., King, G. D., Hu, J., Sciascia, S. A., Candolfi, M., Greengold, D. S., Lowenstein, P. R., and Castro, M. G. (2005) Regulatable gene expression systems for gene therapy applications: progress and future challenges, Mol Ther 12, 189-211.
5. Xiong, W., Goverdhana, S., Sciascia, S. A., Candolfi, M., Zirger, J. M., Barcia, C., Curtin, J. F., King, G. D., Jaita, G., Liu, C., Kroeger, K., Agadjanian, H., MedinaKauwe, L., Palmer, D., Ng, P., Lowenstein, P. R., and Castro, M. G. (2006) Regulatable gutless adenovirus vectors sustain inducible transgene expression in the brain in the presence of an immune response against adenoviruses, J Virol 80, 27-37.
6. Knott, A., Garke, K., Urlinger, S., Guthmann, J., Muller, Y., Thellmann, M., and Hillen, W. (2002) Tetracycline-dependent gene regulation: combinations of transregulators yield a variety of expression windows, Biotechniques 32, 796, 98, 800 passim.
7. Urlinger, S., Baron, U., Thellmann, M., Hasan, M. T., Bujard, H., and Hillen, W. (2000) Exploring the sequence space for tetracycline-dependent transcriptional activators: novel mutations yield expanded range and sensitivity, Proc Natl Acad Sci USA 91, 7963-8.
8. Freundlieb, S., Schirra-Muller, C., and Bujard, H. (1999) A tetracycline controlled activation/repression system with increased potential for gene transfer into mammalian cells, J Gene Med 1, 4-12.
9. Lamartina, S., Silvi, L., Roscilli, G., Casimiro, D., Simon, A. J., Davies, M. E., Shiver, J. W., Rinaudo, C. D., Zampaglione, I., Fattori, E., Colloca, S., Gonzalez Paz, O., Laufer, R., Bujard, H., Cortese, R., Ciliberto, G., and Toniatti, C. (2003) 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 1, 271-80.
10. Candolfi, M., Curtin, J. F., Xiong, W. D., Kroeger, K. M., Liu, C., Rentsendorj, A., Agadjanian, H., Medina-Kauwe, L., Palmer, D., Ng, P., Lowenstein, P. R., and Castro, M. G. (2006) Effective high-capacity gutless adenoviral vectors mediate transgene expression in human glioma cells, Mol Ther 14, 371-81.
11. Candolfi, M., Pluhař, G. E., Kroeger, K., and Puntel, M. C J., Barcia, C., Muhammad, A.G., Xiong, W., Liu, C., Mondkar, S., Kuoy, W., Kang, T., McNeil, EA., Freese, A. B., Ohlfest J. R., Moore, P., Palmer, D. Ng. P., Young, J.D., Lowenstein, P.R., Castro, M.G. (2006) Optimization of adenoviral vector mediated expression in the canine brain in vivo, and in canine glioma cells in vitro., Neurooncology Under Review.
12. Joseph Sambrook, and Russell, D. W. (2001) Molecular Cloning: A Laboratory Manual, Cold Spring Harbor Laboratory Press, Woodbury, NY, USA.
13. Umana, P., Gerdes, C A., Stone, D., Davis, J. R., Ward, D., Castro, M. G., and Lowenstein, P. R. (2001) Efficient FLPe recombinase enables scalable production of helper-dependent adenoviral vectors with negligible helper-virus contamination, Nat Biotechnol 19, 582-5.
14. Maleniak, T. C., Darling, J. L., Lowenstein, P. R., and Castro, M. G. (2001) Adenovirus-mediated expression of HSV1-TK or Fas ligand induces cell death in primary human glioma-derived cell cultures that are resistant to the chemotherapeutic agent CCNU, Cancer Gene Ther 8, 589-98.
15. Ali, S., Curtin, J. F., Zirger, J. M., Xiong, W., King, G. D., Barcia, C., Liu, C., Puntel, M., Goverdhana, S., Lowenstein, P. R., and Castro, M. G. (2004) Inflammatory and anti-glioma effects of an adenovirus expressing human soluble Fms-like tyrosine kinase 3 ligand (hsFlt3L): treatment with hsFlt3L inhibits intracranial glioma progression, Mol Ther 10, 1071-84.
16. Williams, J. C., Stone, D., Smith-Arica, J. R., Morris, I. D., Lowenstein, P. R., and Castro, M. G. (2001) Regulated, adenovirus-mediated delivery of tyrosine hydroxylase suppresses growth of estrogen-induced pituitary prolactinomas, Mol Ther 4, 593-602.
17. Smith-Arica, J. R., Morelli, A. E., Larregina, A. T., Smith, J., Lowenstein, P. R., and Castro, M. G. (2000) Cell-type-specific and regulatable transgenesis in the adult brain: adenovirus-encoded combined transcriptional targeting and inducible transgene expression, Mol Ther 2, 579-87.
18. Southgate, T. D., Kingston, P. A., and Castro, M. G. (2000) Gene Transfer into Neural Cells In Vitro using Adenoviral Vectors, John Wiley & Sons, Inc., New York.
19. Palmer, D., and Ng, P. (2003) Improved system for helper-dependent adenoviral vector production, Mol Ther 8, 846-52.
20. Ng, P., Parks, R. J., Cummings, D. T., Evelegh, C. M., and Graham, F. L. (2000) An enhanced system for construction of adenoviral vectors by the two-plasmid rescue method, Hum Gene Ther 11, 693-9.
21. Li, Y., McCadden, J., Ferrer, F., Kruszewski, M., Carducci, M., Simons, J., and Rodriguez, R. (2002) Prostate-specific expression of the diphtheria toxin A chain (DTA): studies of inducibility and specificity of expression of prostate-specific antigen promoter-driven DT-A adenoviral-mediated gene transfer, Cancer Res 62, 2576-82.
22. Kohno, K., and Uchida, T. (1987) Highly frequent single amino acid substitution in mammalian elongation factor 2 (EF-2) results in expression of resistance to EF-2-ADP-ribosylating toxins, J Biol Chem 262, 12298-305.
23. Puntel, M., Curtin, J. F., Zirger, J. M., Muhammad, A. K., Xiong, W., Liu, C., Hu, J., Kroeger, K. M., Czer, P., Sciascia, S., Mondkar, S., Lowenstein, P. R., and Castro, M. G. (2006) Quantification of high-capacity helper-dependent adenoviral vector genomes in vitro and in vivo, using quantitative TaqMan real-time polymerase chain reaction, Hum Gene Ther 17, 531-44.
24. Gerdes, C A., Castro, M. G., and Lowenstein, P. R. (2000) Strong promoters are the key to highly efficient, noninflammatory and noncytotoxic adenoviral-mediated transgene delivery into the brain in vivo, Mol Ther 2, 330-8.
25. Schiedner, G., Morral, N., Parks, R. J., Wu, Y., Koopmans, S. C., Langston, C., Graham, F. L., Beaudet, A. L., and Kochanek, S. (1998) Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity, Nat Genet 18, 180-3.