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Drug Discov Today. 2007 Jul;12(13-14):553-60. Epub 2007 Jun 28.

Mining the GEMS--a novel platform technology targeting post-transcriptional control mechanisms.

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  • 1PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ 07080, USA.


The physiological levels of many clinically important proteins are regulated through cellular mechanisms that control the stability and translational efficiency of mRNA. These post-transcriptional processes, which play a critical role in the regulation of gene expression, depend on interactions of specific trans-acting factors with sequence elements located within the 5'- and 3'-untranslated regions (UTRs) of an mRNA. A technology platform called GEMS (Gene Expression Modulation by Small-molecules) exploits the interactions of UTR elements with the trans-acting factors, thereby specifically targeting mechanisms of post-transcriptional control. In this review we describe how this technology enables the identification of small-molecules that modulate the levels of proteins involved in disease pathogenesis.

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