Repairing Purkinje cell connections to improve balance in ataxia
Developing Purkinje Cell Synaptic Therapies to Restore Circuit Function in Cerebellar Degeneration
Trying a new approach to restore key nerve-cell connections in the cerebellum to help people with cerebellar degeneration regain balance and coordination.
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 ID | NIH-11248046 on NIH RePORTER |
What this research studies
This project focuses on the Purkinje cells in the cerebellum, which are central to balance and movement. Researchers will use animal models and examine human postmortem brain tissue to map how climbing fiber–Purkinje cell synapses change early in degeneration. They will test interventions that modify these synapses while monitoring cerebellar alpha rhythms and movement to see whether coordination improves. The work is preclinical groundwork intended to guide possible future treatments for people with ataxia.
Who could benefit from this research
Good fit: Ideal candidates would be people with cerebellar degeneration or progressive ataxia, particularly those in earlier stages when synaptic changes are present.
Not a fit: People whose movement problems come from non-cerebellar causes or who have very advanced Purkinje cell loss may not benefit from synapse-targeted approaches.
Why it matters
Potential benefit: If successful, the work could lead to therapies that restore cerebellar circuit function and reduce imbalance, falls, and loss of coordination in people with ataxia.
How similar studies have performed: Animal studies and postmortem human work link Purkinje synapse changes to ataxia, but targeted synaptic therapies are largely novel and have not yet been proven in humans.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Kuo, Sheng-Han — Columbia University Health Sciences
- Study coordinator: Kuo, Sheng-Han
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.