Rhes‑SUMO pathway's role in Huntington's disease
Rhes-SUMO Pathway in Huntington Disease
This work looks at whether the Rhes‑SUMO protein system increases toxic huntingtin and helps it spread in the brain, which may explain early striatal damage in people with Huntington's disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Florida Atlantic University NIH-funded |
| Lab location | 1 site (Boca Raton, United States) |
| Project ID | NIH-11299742 on NIH RePORTER |
What this research studies
Researchers are focusing on two molecules — Rhes and SUMO1 — that seem to change levels and spread of mutant huntingtin protein in the brain. They will use cell models and a genetically accurate mouse model of HD to track how Rhes and SUMO1 affect huntingtin movement between neurons and its buildup. The team will test whether reducing SUMO signaling or removing SUMO1 lowers toxic huntingtin and improves brain structure and behavior in the animal model. Results are intended to point to targets that could be used to slow striatal damage and limit spread of the disease.
Who could benefit from this research
Good fit: People who carry the HTT gene mutation — either diagnosed with Huntington's disease or pre-symptomatic carriers — are the patients most relevant to this research.
Not a fit: People without the HTT mutation or those looking for immediate clinical treatments are unlikely to benefit directly because this is laboratory and animal-focused work.
Why it matters
Potential benefit: If successful, this work could point to new ways to lower toxic huntingtin or block its spread, potentially leading to therapies that slow or prevent Huntington's progression.
How similar studies have performed: Previous preclinical studies have shown Rhes interacts with mutant huntingtin and that reducing SUMO1 can improve outcomes in HD mouse models, so there is encouraging lab-based evidence but no proven human therapies yet.
Where this research is happening
Boca Raton, United States
- Florida Atlantic University — Boca Raton, United States (Active)
Researchers
- Principal investigator: Subramaniam, Srinivasa — Florida Atlantic University
- Study coordinator: Subramaniam, Srinivasa
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.