Binding to RNA is not enough-changing its shape is what makes a drug work, study reveals
Phys.org • • science
Key Points:
- RNA molecules serve as messengers between DNA and protein production and have diverse regulatory roles, making them important targets for drug design.
- Research by Danny Incarnato and his team reveals that small molecules binding to RNA rarely affect its function, whereas those that alter RNA structure have a more significant impact.
- Despite substantial investments, only one mRNA-targeting small molecule drug, risdiplam, has reached the market, highlighting gaps in understanding effective RNA-targeting drugs.
- The study demonstrates that RNA can adopt multiple shapes, and the cancer drug mitoxantrone stabilizes a specific RNA conformation, influencing its function.
- Incarnato suggests that future RNA-targeting drug development should focus not only on molecules that bind RNA but also on those that can modulate its structural folding.
