Editing proteins to reduce harmful effects in prion disease.
Official title: Therapeutic editing to lower PrP in prion disease: Administrative Core
Funded at Broad Institute, INC. · NIH-11085945 · Data updated 2026-11-12
This study is looking at new ways to reduce a harmful protein linked to prion diseases, which could help improve treatment options and quality of life for patients dealing with these tough conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | May 17, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $336,305 |
| NIH project number | 5U19NS132315-03 |
| Project ID | NIH-11085945 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with prion diseases or those at risk of developing these conditions.
✕Less likely to help
Patients with other neurodegenerative disorders unrelated to prion diseases may not receive any benefit from this research.
What this research studies
This research focuses on developing therapeutic strategies to lower the levels of prion protein (PrP) associated with prion diseases, which are neurodegenerative disorders. The approach involves innovative editing techniques aimed at modifying the expression of PrP to potentially halt or reverse disease progression. Patients may benefit from advancements in treatment options that target the underlying mechanisms of these diseases, improving their quality of life and health outcomes.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the management of prion diseases.
How similar studies have gone: While the specific approach of therapeutic editing for prion diseases is novel, similar strategies have shown promise in other neurodegenerative conditions.
Where this research is happening
-
Broad Institute, INC.
Cambridge, United States Active
Researchers
- Principal investigator: Vallabh, Sonia Minikel — Broad Institute, INC.
- Study coordinator: Vallabh, Sonia Minikel
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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.