Creating a special mouse model to study ataxin-1's role in diseases.
Development of a conditional ataxin-1 knockout mouse line
['FUNDING_R03'] · EAST CAROLINA UNIVERSITY · NIH-10778628
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
| Phase | ['FUNDING_R03'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | EAST CAROLINA UNIVERSITY (nih funded) |
| Locations | 1 site (GREENVILLE, UNITED STATES) |
| Trial ID | NIH-10778628 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals affected by Alzheimer's disease or related neurodegenerative disorders.
Not a fit: Patients with conditions unrelated to ataxin-1 or those not affected by neurodegenerative diseases may not benefit from this research.
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 performed: 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
GREENVILLE, UNITED STATES
- EAST CAROLINA UNIVERSITY — GREENVILLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: DIDONNA, ALESSANDRO — EAST CAROLINA UNIVERSITY
- Study coordinator: DIDONNA, ALESSANDRO
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease