Designing drugs that target RNA repeat expansions
In Silico Drug Design Targeting RNA Repeat Expansions
['FUNDING_R15'] · FLORIDA ATLANTIC UNIVERSITY · NIH-10439166
This study is working on new computer tools to see how tiny molecules connect with RNA structures that can cause genetic disorders, like Fragile X Syndrome and Myotonic Dystrophy, to help design better treatments for these conditions.
Quick facts
| Phase | ['FUNDING_R15'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | FLORIDA ATLANTIC UNIVERSITY (nih funded) |
| Locations | 1 site (BOCA RATON, UNITED STATES) |
| Trial ID | NIH-10439166 on ClinicalTrials.gov |
What this research studies
This research focuses on developing advanced computational tools to understand how small molecules interact with RNA structures that are linked to genetic disorders. By utilizing a novel physics-based method, the project aims to predict how these molecules bind to dynamic RNA loops and optimize their properties for better drug design. The ultimate goal is to create effective pharmacotherapies for conditions caused by RNA repeat expansions, such as Fragile X Syndrome and Myotonic Dystrophy.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with genetic disorders like Fragile X Syndrome, Myotonic Dystrophy, or Huntington's disease.
Not a fit: Patients with genetic disorders not related to RNA repeat expansions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new and effective treatments for genetic disorders associated with RNA repeat expansions.
How similar studies have performed: Previous research has shown promise in using computational methods for drug design targeting RNA, indicating potential for success in this approach.
Where this research is happening
BOCA RATON, UNITED STATES
- FLORIDA ATLANTIC UNIVERSITY — BOCA RATON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: YILDIRIM, ILYAS — FLORIDA ATLANTIC UNIVERSITY
- Study coordinator: YILDIRIM, ILYAS
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Disease