Understanding RAN Proteins in Huntington's Disease and Spinocebellar Ataxias
Contribution of RAN proteins to HD, SCA3 other CAG.CTG expansion diseases
This research explores how certain proteins, called RAN proteins, contribute to conditions like Huntington's disease and various spinocerebellar ataxias, with the goal of finding new ways to help patients.
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-11144595 on NIH RePORTER |
What this research studies
Our team discovered RAN proteins in 2011, and since then, we've learned that these proteins build up in people with nine different expansion disorders, including Huntington's disease and spinocerebellar ataxia. We are now working to understand if these RAN proteins are harmful and if we can reduce their levels to lessen disease symptoms. We are developing new tools, including gene therapy (AAV) and small molecule approaches, to block the production of these RAN proteins. Our hope is that by targeting these proteins, we can find new treatments for these challenging conditions.
Who could benefit from this research
Good fit: This foundational research is relevant to individuals living with Huntington's disease, spinocerebellar ataxia type 3 (SCA3), and other related CAG·CTG expansion disorders.
Not a fit: Patients whose conditions are not related to CAG·CTG repeat expansion disorders or RAN protein accumulation would likely not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that reduce harmful RAN proteins, potentially slowing or stopping the progression of Huntington's disease and various spinocerebellar ataxias.
How similar studies have performed: Our team and others have previously shown that RAN proteins accumulate in disease-relevant human tissues, building a foundation for this current approach to target them.
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.