Investigating the role of RAN proteins in neurodegenerative diseases
Contribution of RAN proteins to HD, SCA3 other CAG.CTG expansion diseases
This study is looking at how certain proteins might play a role in diseases like Huntington's and spinocerebellar ataxias, and it aims to find ways to reduce these proteins in cells and mice to help discover new treatments for these conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project ID | NIH-10986050 on NIH RePORTER |
What this research studies
This research focuses on understanding how RAN proteins contribute to various neurodegenerative diseases, particularly those caused by CAG and CTG repeat expansions, such as Huntington's disease and spinocerebellar ataxias. The team will explore the accumulation of these proteins in human tissues and utilize advanced techniques, including AAV and small molecules, to inhibit their production. By testing these approaches in both human-derived cells and mouse models, the research aims to uncover common mechanisms of toxicity and potential therapeutic strategies.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with Huntington's disease or spinocerebellar ataxias caused by CAG/CTG repeat expansions.
Not a fit: Patients with neurodegenerative diseases not associated with CAG/CTG repeat expansions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that reduce the harmful effects of RAN proteins in patients with neurodegenerative diseases.
How similar studies have performed: Previous research has shown promising results in targeting RAN proteins in related disorders, indicating potential for success in this approach.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Ranum, Laura P.w — University of Florida
- Study coordinator: Ranum, Laura P.w
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.