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-11177309 · Data updated 2026-11-12
This study is looking for new treatment options for people with spinocerebellar ataxias, a group of rare disorders that affect balance and coordination, by testing existing medications and natural products to see if they can help reduce the problems caused by specific 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) | $79,387 |
| NIH project number | 3R01NS135254-02S2 |
| Project ID | NIH-11177309 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 caused by 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 caused by genetic mutations that lead to progressive loss of coordination and balance. The researchers aim 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, they will screen for existing FDA-approved drugs and natural products that may reduce the harmful effects of these mutations. This approach could lead to new treatment options for patients suffering from these debilitating conditions.
Why it matters
Potential benefit: If successful, this research could provide new therapeutic options for patients with spinocerebellar ataxias, potentially improving their quality of life and slowing disease progression.
How similar studies have gone: Previous research has shown promise in targeting similar genetic mutations with small molecules, indicating potential for success in this novel 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.