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Updated: Jun 21, 2026
09:44
High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
Published on: March 3, 2015
Targeting post-transcriptional control for drug discovery
Stuart W Peltz1, Ellen M Welch, Christopher R Trotta
1PTC Therapeutics Inc, South Plainfield, NJ 07080, USA. speltz@ptcbio.com
RNA Biology
|July 4, 2009
Summary
Researchers developed novel drug screening technologies targeting RNA regulation to control gene expression. These methods identified drug candidates currently in clinical trials for unmet medical needs, highlighting the potential of post-transcriptional control.
Area of Science:
- Molecular Biology
- Genetics
- Drug Discovery
Background:
- Post-transcriptional regulation significantly impacts gene expression through RNA interactions.
- This regulation offers numerous targets for small molecule drugs to modulate protein accumulation.
Purpose of the Study:
- To describe two drug screening technologies targeting post-transcriptional regulation of specific messenger RNAs (mRNAs).
- To illustrate the therapeutic potential of targeting post-transcriptional control for diseases with unmet needs.
Main Methods:
- Gene Expression Modulation by Small Molecules (GEMS) technology uses 5' and 3' untranslated regions (UTRs) to identify compounds reducing protein production.
- A second method uses nonsense-containing mRNAs to screen for compounds promoting therapeutic nonsense suppression.
Main Results:
- Both screening programs successfully identified drug candidates.
- These candidates are currently undergoing clinical testing for human diseases.
Conclusions:
- Targeting post-transcriptional control represents a promising therapeutic strategy.
- The developed technologies demonstrate significant potential for treating diseases with high unmet clinical needs.
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