Creating a special mouse model to study ataxin-1's role in diseases.
Official title: Development of a conditional ataxin-1 knockout mouse line
Funded at East Carolina University · NIH-10778628 · Data updated 2026-11-12
This study is creating a special mouse model to learn more about a protein called ataxin-1, which is important for brain health and may be linked to diseases like Alzheimer's, so we can better understand how it works and help people with these conditions.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | East Carolina University NIH-funded |
| Lab location | 1 site (Greenville, United States) |
| Project start | February 15, 2023 |
| Project end | January 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $75,500 |
| NIH project number | 5R03NS131908-02 |
| Project ID | NIH-10778628 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals affected by Alzheimer's disease or related neurodegenerative disorders.
✕Less likely to help
Patients with conditions unrelated to ataxin-1 or those not affected by neurodegenerative diseases may not benefit from this research.
What this research studies
This research focuses on developing a conditional knockout mouse model for ataxin-1, a protein involved in various neurological processes, including Alzheimer's disease. By using advanced techniques like CRISPR/Cas9 and the Cre/Lox system, the researchers aim to better understand how ataxin-1 functions in different cell types and stages of development. This model will allow for a more precise investigation of ataxin-1's role in both neurodegenerative diseases and immune responses in the central nervous system. Patients may benefit from insights gained about ataxin-1's involvement in conditions like Alzheimer's disease.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for Alzheimer's disease and other neurodegenerative conditions.
How similar studies have gone: Previous research has shown promise in using conditional knockout models to study gene functions in various diseases, indicating potential success for this approach.
Where this research is happening
-
East Carolina University
Greenville, United States Active
Researchers
- Principal investigator: Didonna, Alessandro — East Carolina University
- Study coordinator: Didonna, Alessandro
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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.