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Updated: Jul 13, 2026

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
Published on: March 3, 2015
Mining the GEMS--a novel platform technology targeting post-transcriptional control mechanisms
Anuradha Bhattacharyya1, Christopher R Trotta, Stuart W Peltz
1PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ 07080, USA. abhattacharyya@ptcbio.com
Abstract:
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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