Designing drugs that target RNA repeat expansions
Official title: In Silico Drug Design Targeting RNA Repeat Expansions
Funded at Florida Atlantic University · NIH-10796593 · Data updated 2026-11-12
This study is working on creating new drugs that can better connect with RNA structures linked to genetic disorders like Fragile X Syndrome and Huntington’s disease, with the goal of finding better treatment options for people living with these conditions.
Quick facts
| Grant type | R15 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Florida Atlantic University NIH-funded |
| Lab location | 1 site (Boca Raton, United States) |
| Project start | April 1, 2022 |
| Project end | March 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $90,396 |
| NIH project number | 3R15GM146199-01S1 |
| Project ID | NIH-10796593 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with genetic disorders such as Fragile X Syndrome, Myotonic Dystrophy, or Huntington’s disease.
✕Less likely to help
Patients with disorders not related to RNA repeat expansions may not receive any benefit from this research.
What this research studies
This research focuses on developing advanced computational tools to design drugs that can effectively interact with RNA structures involved in various genetic disorders. By utilizing a novel physics-based method, the project aims to predict how small molecules bind to dynamic RNA loops, which are crucial in diseases like Fragile X Syndrome and Huntington’s disease. The researchers will optimize these small molecules by modifying their functional groups to enhance their binding properties. Ultimately, this work seeks to create new pharmacotherapies that target RNA repeat expansions, potentially leading to better treatment options for affected patients.
Why it matters
Potential benefit: If successful, this research could lead to the development of new drugs that specifically target and treat genetic disorders caused by RNA repeat expansions.
How similar studies have gone: While the approach of using computational tools for drug design is established, the specific focus on RNA repeat expansions represents a novel application that has not been extensively tested.
Where this research is happening
-
Florida Atlantic University
Boca Raton, United States Active
Researchers
- Principal investigator: Yildirim, Ilyas — Florida Atlantic University
- Study coordinator: Yildirim, Ilyas
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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.