Understanding and slowing the brain changes in Spinocerebellar Ataxia Type 1
Official title: Molecular Studies of Spinocerebellar Ataxia Type 1
Funded at Baylor College of Medicine · NIH-11352582 · Data updated 2026-11-12
Researchers are studying how a gene mistake causes brain cells to accumulate harmful proteins in Spinocerebellar Ataxia type 1 and what that means for people who might develop this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | September 1, 1989 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $401,250 |
| NIH project number | 5R01NS027699-38 |
| Project ID | NIH-11352582 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with SCA1 or related spinocerebellar ataxias who might participate in future translational studies or benefit from proteopathy-focused therapies.
✕Less likely to help
Patients without SCA1 or related ataxias or those not aligning with potential future therapeutic paths may not directly benefit.
What this research studies
This project looks at why certain brain regions are affected first in spinocerebellar ataxias and how reducing the disease-causing protein in a mouse model can improve cerebellar function. By comparing SCA1 with other neurodegenerative diseases, the team hopes to uncover targets that could help preserve coordination and brain health in people with ataxia. The work combines genetic and molecular approaches to understand where and why the disease starts, with the goal of guiding future therapies. Results could inform strategies to prevent progression and protect brain regions crucial for movement and thinking.
Why it matters
Potential benefit: If successful, the research could guide new approaches to slow or stop disease progression and help people with SCA1 maintain balance and cognitive functions longer.
How similar studies have gone: Previous work shows that lowering disease-related proteins can improve specific brain functions in models, but regional vulnerability requires further study.
Where this research is happening
-
Baylor College of Medicine
Houston, United States Active
Researchers
- Principal investigator: Zoghbi, Huda Y — Baylor College of Medicine
- Study coordinator: Zoghbi, Huda Y
Related research
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Targeting FICD to Help Spinocerebellar Ataxia Type 3 (SCA3)
A brain-focused project tests whether blocking FICD, a protein in cells’ ER quality system, can slow or prevent damage in people (via animal and cell studies) who have spinocerebellar ataxia type 3.
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Understanding how unstable GAA repeats affect Friedreich’s ataxia progression
A study in adults and children with Friedreich’s ataxia will look at how changes in GAA repeat length inside the FXN gene relate to the disease’s start and progression.
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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.