Mining the GEMS--a novel platform technology targeting post-transcriptional control mechanisms.
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.
Bhattacharyya
Anuradha
A
PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ 07080, USA. abhattacharyya@ptcbio.com
Trotta
Christopher R
CR
Peltz
Stuart W
SW
D020413:3' Untranslated Regions; D020121:5' Untranslated Regions; D015195:Drug Design; D005786:Gene Expression Regulation; D006801:Humans; D012323:RNA Processing, Post-Transcriptional; D012333:RNA, Messenger / Q000235:genetics / Q000502:physiology; D054852:Small Molecule Libraries; D015534:Trans-Activators / Q000235:genetics / Q000378:metabolism
D020413:3' Untranslated Regions; D020121:5' Untranslated Regions; D012333:RNA, Messenger; D054852:Small Molecule Libraries; D015534:Trans-Activators