Abstract
It is feasible to restrict transgene expression to a tissue or region in need of therapy by using promoters that respond to focusable physical stimuli. The most extensively investigated promoters of this type are radiation-inducible promoters and heat shock protein gene promoters that can be activated by directed, transient heat. Temporal regulation of transgenes can be achieved by various two- or three-component gene switches that are triggered by an appropriate small molecule inducer. The most commonly considered gene switches that are reviewed herein are based on small moleculeresponsive transactivators derived from bacterial tetracycline repressor, insect or mammalian steroid receptors, or mammalian FKBP12/FRAP. A new generation of gene switches combines a heat shock protein gene promoter and a small molecule-responsive gene switch and can provide for both spatial and temporal regulation of transgene activity.
Keywords: Gene therapy, gene switch, transcriptional targeting, spatial regulation, temporal regulation
Current Gene Therapy
Title: Regulatable Gene Expression Systems for Gene Therapy
Volume: 6 Issue: 4
Author(s): Nuria Vilaboa and Richard Voellmy
Affiliation:
Keywords: Gene therapy, gene switch, transcriptional targeting, spatial regulation, temporal regulation
Abstract: It is feasible to restrict transgene expression to a tissue or region in need of therapy by using promoters that respond to focusable physical stimuli. The most extensively investigated promoters of this type are radiation-inducible promoters and heat shock protein gene promoters that can be activated by directed, transient heat. Temporal regulation of transgenes can be achieved by various two- or three-component gene switches that are triggered by an appropriate small molecule inducer. The most commonly considered gene switches that are reviewed herein are based on small moleculeresponsive transactivators derived from bacterial tetracycline repressor, insect or mammalian steroid receptors, or mammalian FKBP12/FRAP. A new generation of gene switches combines a heat shock protein gene promoter and a small molecule-responsive gene switch and can provide for both spatial and temporal regulation of transgene activity.
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Cite this article as:
Vilaboa Nuria and Voellmy Richard, Regulatable Gene Expression Systems for Gene Therapy, Current Gene Therapy 2006; 6 (4) . https://dx.doi.org/10.2174/156652306777934829
DOI https://dx.doi.org/10.2174/156652306777934829 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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