Restoring brain function in patients with cerebellar degeneration
Official title: Developing Purkinje Cell Synaptic Therapies to Restore Circuit Function in Cerebellar Degeneration
Funded at Columbia University Health Sciences · NIH-11050518 · Data updated 2026-11-12
This study is looking for ways to help people with ataxia, a condition that makes it hard to balance and control movements, by exploring how to fix the brain cells that are damaged in this condition, with hopes of finding new treatments that can really make a difference.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | January 1, 2025 |
| Project end | December 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $605,523 |
| NIH project number | 1R01NS136686-01A1 |
| Project ID | NIH-11050518 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cerebellar degeneration who experience ataxia symptoms.
✕Less likely to help
Patients with ataxia symptoms caused by conditions unrelated to cerebellar degeneration may not benefit from this research.
What this research studies
This research focuses on developing therapies to restore the function of brain circuits affected by cerebellar degeneration, which leads to severe ataxia symptoms like imbalance and loss of movement control. By studying the changes in Purkinje cells, the main neurons in the cerebellum, the research aims to identify how to intervene at specific stages of degeneration. The approach involves using animal models to understand the effects of synaptic interventions on cerebellar function and ataxia symptoms, with the goal of translating these findings into potential treatments for patients. If successful, this research could pave the way for new therapies that directly address the underlying causes of ataxia.
Why it matters
Potential benefit: If successful, this research could lead to effective therapies that significantly improve movement control and reduce falls in patients with cerebellar degeneration.
How similar studies have gone: Previous research has shown promise in targeting synaptic changes in animal models, suggesting that this approach could be effective, although it remains largely untested in human subjects.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Kuo, Sheng-Han — Columbia University Health Sciences
- Study coordinator: Kuo, Sheng-Han
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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.