Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Regulatable Gene Expression Systems for Gene Therapy

Author(s): Nuria Vilaboa, Richard Voellmy.

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

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Article Details

VOLUME: 6
ISSUE: 4
Year: 2006
Page: [421 - 438]
Pages: 18
DOI: 10.2174/156652306777934829
Price: $58