Deciphering RNA-Related Blood-Brain Barrier Breakdown in C9orf72-Linked Frontotemporal Dementia
Official title: Deciphering RNA-Mediated Blood-Brain Barrier Dysfunction in C9orf72-Linked Frontotemporal Dementia (FTD)
Funded at University of Vermont & St Agric College · NIH-11335687 · Data updated 2026-11-12
A patient-focused look at how harmful RNA buildup in a gene linked to frontotemporal dementia may weaken the brain’s protective barrier and what that could mean for future therapies.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Vermont & St Agric College NIH-funded |
| Lab location | 1 site (Burlington, United States) |
| Project start | December 1, 2020 |
| Project end | March 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $282,439 |
| NIH project number | 2P20GM135007-06A1 |
| Project ID | NIH-11335687 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with or at risk for C9orf72-related frontotemporal dementia or those who can provide related biological samples for study.
✕Less likely to help
Individuals without C9orf72-related risk or related samples may not directly benefit from this research.
What this research studies
This project studies how specific RNA toxicity from the C9orf72 gene might disrupt the blood-brain barrier, a critical shield protecting the brain. Researchers will use human-relevant models to explore how astrocytes (brain support cells) contribute to barrier stability and test a new RNA-targeting tool to fix the problem. The goal is to map the molecular steps from RNA toxicity to barrier dysfunction and neurodegeneration so future treatments can target these steps. The work combines advanced 3-D models and molecular approaches to connect cellular changes with potential therapies.
Why it matters
Potential benefit: If successful, the work could guide new therapies that restore brain barrier function and slow or prevent progression of C9orf72-related frontotemporal dementia.
How similar studies have gone: Preliminary data suggest that targeting RNA toxicity can restore astrocyte support and vascular stability, supporting potential for similar approaches.
Where this research is happening
-
University of Vermont & St Agric College
Burlington, United States Active
Researchers
- Principal investigator: Morelli, Kathryn H — University of Vermont & St Agric College
- Study coordinator: Morelli, Kathryn H
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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.