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Regulated gene expression systems

Gene Therapy volume 7, pages 120–125 (2000)Cite this article

Abstract

Most gene therapy research to date has focused on solving the delivery problem – getting genes efficiently and stably into target cells and tissues. Those working on systems for regulating the expression of genes once delivered have often been accused of trying to run before they can walk. Yet regulation is likely to be essential if gene therapy is to realize its full potential as a mainstream clinical option for delivering proteins. Dramatic progress has been made in designing and testing systems in which expression is controlled by orally active drugs. The next few years should see the first clinical trials of drug-regulated gene therapies.

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Acknowledgements

I thank all my ARIAD colleagues and collaborators for discussions and enlightenment, and Vic Rivera for critical review of the manuscript.

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  1. ARIAD Gene Therapeutics Inc, 26 Landsdowne Street, Cambridge, MA 02139, USA

    T Clackson

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  1. T Clackson
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Clackson, T. Regulated gene expression systems. Gene Ther 7, 120–125 (2000). https://doi.org/10.1038/sj.gt.3301120

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