Investigating small molecule treatments for spinocerebellar ataxias caused by CAG expansion mutations
Official title: Alternative Splicing and Development of Small Molecule Therapeutics in CAG Expansion Spinocerebellar Ataxias
Funded at State University of New York at Albany · NIH-10993152 · Data updated 2026-11-12
This study is looking for new treatments for spinocerebellar ataxias (SCAs), which are conditions that affect coordination and balance, by testing existing medications and natural products to see if they can help reduce harmful effects caused by certain genetic mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University of New York at Albany NIH-funded |
| Lab location | 1 site (Albany, United States) |
| Project start | December 1, 2023 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $516,372 |
| NIH project number | 5R01NS135254-02 |
| Project ID | NIH-10993152 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with spinocerebellar ataxias caused by CAG expansion mutations.
✕Less likely to help
Patients with spinocerebellar ataxias not associated with CAG expansion mutations may not benefit from this research.
What this research studies
This research focuses on spinocerebellar ataxias (SCAs), a group of rare neurological disorders that lead to progressive loss of coordination and balance. The study aims to develop small molecule therapeutics that target CAG expansion mutations, which are common in several types of SCAs. By using a specialized cell line that mimics these mutations, researchers will screen for existing FDA-approved drugs and natural products that may reduce harmful RNA levels associated with these conditions. The goal is to find effective treatments that could benefit multiple SCA types simultaneously.
Why it matters
Potential benefit: If successful, this research could lead to new treatment options that improve coordination and quality of life for patients with spinocerebellar ataxias.
How similar studies have gone: Previous research has shown promise in targeting similar genetic mutations with small molecules, suggesting potential for success in this approach.
Where this research is happening
-
State University of New York at Albany
Albany, United States Active
Researchers
- Principal investigator: Berglund, Andrew — State University of New York at Albany
- Study coordinator: Berglund, Andrew
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.