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Updated: Aug 8, 2026
09:44
High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
Published on: March 3, 2015
Gene expression as a target for new drug discovery
A Heguy1, A A Stewart, J D Haley
1Oncogene Science, Inc., Uniondale, NY 11553, USA.
Gene Expression
|January 1, 1995
Summary
Gene transcription modulation offers a novel drug discovery approach. This method identifies compounds that regulate gene expression for therapeutic benefits, including treating sickle cell disease and beta-thalassemia.
Area of Science:
- Molecular Biology
- Pharmacology
- Drug Discovery
Background:
- Gene transcription is explored as a novel strategy for drug discovery.
- The objective is to find small molecules that precisely control gene expression.
Purpose of the Study:
- To introduce transcription-based pharmaceuticals as a new therapeutic modality.
- To demonstrate the application of this approach in drug discovery programs.
- To highlight its potential for treating genetic blood disorders like sickle cell disease and beta-thalassemia.
Main Methods:
- Focus on identifying small molecular weight compounds.
- Modulating gene expression to alter protein product concentration.
- Developing transcription-based drug discovery programs.
Main Results:
- Established a 7-year research effort in gene transcription for drug discovery.
- Identified the potential of transcriptional modulation to replace recombinant proteins.
- Demonstrated a novel approach to manipulate key gene targets.
Conclusions:
- Transcription-based pharmaceuticals represent a promising new class of drugs.
- This approach offers a versatile strategy for targeting various diseases.
- The research program shows potential for developing new treatments for sickle cell disease and beta-thalassemia.
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