Regulatable Gene Expression in Gene Therapy

Fabio Rossi, University of British Columbia, Vancouver, British Columbia, Canada

Published online: January 2006

DOI: 10.1038/npg.els.0005748

The regulation of exogenous gene expression may soon be a requirement for gene therapy approaches. Several systems promise to fulfill requirements for clinical applications.

Keywords: inducible gene expression; tetracycline; ecdyson; rapamycin/rapalogs; RU486; gene therapy; iathrogenic effects

 References
    Gossen M and Bujard H (1992) Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proceedings of the National Academy of Sciences of the United States of America 89(12): 5547–5551.
    Gossen M, Freundlieb S, Bender G, et al. (1995) Transcriptional activation by tetracyclines in mammalian cells. Science 268: 1766–1769.
    Liberles SD, Diver ST, Diver, Austin DJ, et al. (1997) Inducible gene expression and protein translocation using nontoxic ligands identified by a mammalian three-hybrid screen. Proceedings of the National Academy of Sciences of the United States of America 94(15): 7825–7830.
    Magari SR, Rivera VM, Iuliucci JD, et al. (1997) Pharmacologic control of a humanized gene therapy system implanted into nude mice. Journal of Clinical Investigation 100: 2865–2872.
    No D, Yao TP, Evans RM, et al. (1996) Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proceedings of the National Academy of Sciences of the United States of America 93(8): 3346–3351.
    Pomerantz JL, Sharp PA, Pabo CO, et al. (1995) Structure-based design of transcription factors. Science 267: 93–96.
    Rivera VM, Clackson T, Natesan S, et al. (1996) A humanized system for pharmacologic control of gene expression. Nature Medicine 2(9): 1028–1032.
    Rivera VM, Wang X, Wardwell S, et al. (2000) Regulation of protein secretion through controlled aggregation in the endoplasmic reticulum. Science 287: 826–830.
    Rossi FM, Guicherit OM, Spicher A, et al. (1998) Tetracycline-regulatable factors with distinct dimerization domains allow reversible growth inhibition by p16. Nature Genetics 20(4): 389–393.
    Saez E, Nelson MC, Eshelman B, et al. (2000) Identification of ligands and coligands for the ecdysone-regulated gene switch. Proceedings of the National Academy of Sciences of the United States of America 97(26): 14512–14517.
    Vegeto E, Allan GF, Schrader WT et al. (1992) The mechanism of RU486 antagonism is dependent on the conformation of the carboxy-terminal tail of the human progesterone receptor. Cell 69: 703–713.
    Wang Y, Xu J, Pierson B, O'Malley BW and Tsai SY (1997) Positive and negative regulation of gene expression in eukaryotic cells with an inducible transcriptional regulator. Gene Therapy 4(5): 432–441.
 Further Reading
    Corbel SY and Rossi FM (2002) Latest developments and in vivo use of the Tet system: ex vivo and in vivo delivery of tetracycline-regulated genes. Current Opinion in Biotechnology 13(5): 448–452.
    Weber W, Fux C, Daoud-el Baba M, et al. (2002) Macrolide-based transgene control in mammalian cells and mice. Nature Biotechnology 20(9): 901–907.

Related Sub-Topics:

Techniques and Tools in Clinical Genetics | Gene Therapy | Transcriptional Regulation
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Article Title: Regulatable Gene Expression in Gene Therapy
 
How to Cite close
Rossi, Fabio(Jan 2006) Regulatable Gene Expression in Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. http://www.els.net [doi: 10.1038/npg.els.0005748]