Understanding new proteins in ALS, FTD, and SBMA to find common treatments
Novel repeat associated non-AUG (RAN) proteins in sALS sFTD and SBMA: shared pathological features and unifying therapeutic opportunities
This research explores how unusual proteins might contribute to diseases like ALS, FTD, and SBMA, hoping to uncover 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-10695461 on NIH RePORTER |
What this research studies
Many people with ALS and FTD have forms of the disease where the genetic cause is not yet known. This project looks at specific genetic changes that can lead to unusual proteins building up in the brain and spinal cord, which are seen in diseases like spinal-bulbar muscular atrophy (SBMA). We believe similar unusual proteins might also play a role in sporadic ALS and FTD. By identifying these proteins and understanding how they cause damage, we hope to find common features across these conditions. This knowledge could then lead to new treatment options that work for a wider range of patients.
Who could benefit from this research
Good fit: This foundational research is relevant to patients diagnosed with sporadic Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), or Spinal-Bulbar Muscular Atrophy (SBMA).
Not a fit: Patients whose conditions are not related to the specific protein mechanisms being investigated in ALS, FTD, or SBMA may not directly benefit from this particular line of research.
Why it matters
Potential benefit: If successful, this work could identify new targets for therapies that address the underlying causes of sporadic ALS, FTD, and SBMA.
How similar studies have performed: The concept of repeat associated non-AUG (RAN) translation and the accumulation of novel RAN proteins has been observed in other expansion disorders, suggesting a promising avenue for this research.
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.