Regulatable gene expression systems for gene therapy.

Nuria Vilaboa; Richard Voellmy

doi:10.2174/156652306777934829

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Regulatable gene expression systems for gene therapy.

Nuria Vilaboa·Richard Voellmy

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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 molecule-responsive 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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Animals Gene Expression Regulation Gene Targeting Genetic Therapy Humans Journal Article Research Support, Non-U.S. Gov't Review

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Nuria Vilaboa, Richard Voellmy. 2006. Regulatable gene expression systems for gene therapy.. https://doi.org/10.2174/156652306777934829

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