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Splice-switching small molecules: A new therapeutic approach to modulate gene expression
Andrea Taladriz-Sender1, Emma Campbell1, Glenn A Burley1
1Department of Pure and Applied Chemistry, University of Strathclyde. Thomas Graham Building, 295 Cathedral Street, Glasgow G1 1XL, United Kingdom.
Methods (San Diego, Calif.)
|June 17, 2019
Summary
Small molecules that alter RNA splicing offer therapeutic potential. Understanding how these splice-switching molecules achieve selectivity and their mechanisms of action is crucial for drug development.
Area of Science:
- Molecular Biology
- Drug Discovery
- RNA Therapeutics
Background:
- Alternative RNA splicing is a key regulatory mechanism in gene expression.
- Dysregulation of RNA splicing is implicated in various diseases.
- Small molecules offer a promising avenue for modulating aberrant splicing events.
Purpose of the Study:
- To review the current advancements in splice-switching small molecules.
- To elucidate the molecular determinants of splice-switching selectivity.
- To compare the mechanisms of action of different splice-switching small molecules.
Main Methods:
- Literature review of current research on splice-switching small molecules.
- Analysis of reported small molecules targeting RNA splicing.
- Comparative analysis of the modes of action for selected small molecules.
Main Results:
- Small molecules can effectively manipulate alternative RNA splicing.
- Key molecular determinants influencing splice-switching selectivity have been identified.
- Distinct mechanisms of action exist among different splice-switching small molecules.
Conclusions:
- Small molecule-mediated RNA splicing modulation is a promising therapeutic strategy.
- Further research into selectivity determinants and mechanisms is essential for optimizing drug development.
- This review provides a comprehensive overview of the current landscape of splice-switching small molecules.
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