Understanding Brain Changes in Spinocerebellar Ataxia Type 1
Official title: Molecular Studies of Spinocerebellar Ataxia Type 1
Funded at Baylor College of Medicine · NIH-11123147 · Data updated 2026-11-12
This project aims to understand why diseases like Spinocerebellar Ataxia Type 1 and Alzheimer's disease first affect specific parts of the brain.
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 | 2025 |
| Award (this year) | $401,250 |
| NIH project number | 5R01NS027699-37 |
| Project ID | NIH-11123147 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with neurodegenerative conditions such as Spinocerebellar Ataxia Type 1, Alzheimer's disease, Huntington's disease, or Parkinson's disease may find this fundamental research relevant to their condition.
✕Less likely to help
Patients without neurodegenerative conditions or those with conditions not related to protein accumulation in the brain would not directly benefit from this specific research.
What this research studies
Many brain diseases, including Huntington's, Alzheimer's, Parkinson's, and Spinocerebellar Ataxia, involve harmful protein buildup. While these proteins are found throughout the body, each disease typically starts damaging one specific brain area before spreading. We are exploring why certain brain regions are more vulnerable, using Spinocerebellar Ataxia Type 1 (SCA1) as a model. Our work with SCA1 helps us learn how the disease progresses from affecting coordination to impacting memory, similar to what happens in Alzheimer's disease.
Why it matters
Potential benefit: This work could lead to new treatments that protect specific brain regions from damage in diseases like SCA1 and Alzheimer's, potentially slowing disease progression.
How similar studies have gone: While many groups are exploring ways to reduce harmful protein levels in these diseases, the specific focus on understanding regional brain vulnerability is a less explored but crucial area.
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
Other NIH-funded projects on the same topics.
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Modeling Ataxia Telangiectasia in a human stem cell model of Purkinje cells
Researchers are creating a patient-derived, human cell model of Purkinje cells to understand why this brain disease causes cerebellar degeneration in people with Ataxia Telangiectasia.
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VEGF-mimetic nanoparticles to help spinocerebellar ataxia type 1
A new, patient-friendly approach is being developed to deliver a VEGF-like therapy using tiny particles that could protect brain cells in spinocerebellar ataxia type 1.
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Understanding early Friedreich ataxia in children to improve how we measure outcomes
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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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Biomarkers to measure how well QTE therapy works for Friedreich's ataxia
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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.