Increasing progranulin levels to treat frontotemporal dementia
Official title: Antisense Oligonucleotides for Progranulin-deficient Frontotemporal Dementia
Funded at Saint Louis University · NIH-11170846 · Data updated 2026-11-12
This study is exploring a new treatment for frontotemporal dementia caused by a specific gene mutation, using a special type of therapy that could help boost a protein in the brain, which might improve symptoms and reduce inflammation.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Saint Louis University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | September 30, 2024 |
| Project end | September 29, 2025 |
| Funding year | 2024 |
| Award (this year) | $75,350 |
| NIH project number | 2R56AG064069-06 |
| Project ID | NIH-11170846 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with frontotemporal dementia due to progranulin mutations.
✕Less likely to help
Patients with frontotemporal dementia not caused by progranulin mutations may not benefit from this research.
What this research studies
This research investigates a new treatment approach for frontotemporal dementia (FTD) caused by mutations in the progranulin gene. The study focuses on using antisense oligonucleotides (ASOs), which are short RNA sequences designed to bind to specific RNA targets, to increase the levels of progranulin in the brain. By blocking microRNA binding sites on the GRN mRNA, the researchers aim to enhance progranulin production and assess its effects on behavioral deficits and neuroinflammation in a mouse model. The findings could provide critical insights into the potential of ASO therapy for patients with FTD.
Why it matters
Potential benefit: If successful, this research could lead to a novel treatment that modifies the progression of frontotemporal dementia, improving quality of life for patients.
How similar studies have gone: Other research has shown promise in using antisense oligonucleotides for neurological disorders, indicating potential success for this approach.
Where this research is happening
-
Saint Louis University
Saint Louis, United States Active
Researchers
- Principal investigator: Nguyen, Andrew D. — Saint Louis University
- Study coordinator: Nguyen, Andrew D.
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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.