Lowering harmful proteins in prion disease
Official title: Therapeutic Editing to Lower PrP in Prion Disease
Funded at Broad Institute, INC. · NIH-11085944 · Data updated 2026-11-12
This study is exploring new ways to lower harmful prion proteins in people with prion diseases, hoping to create treatments that could slow down or even reverse these currently untreatable 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) | $4,896,596 |
| NIH project number | 5U19NS132315-03 |
| Project ID | NIH-11085944 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would be individuals diagnosed with prion diseases or those at high risk of developing them.
✕Less likely to help
Patients with prion diseases that are not amenable to genetic or protein-targeting therapies may not benefit from this research.
What this research studies
This research focuses on developing therapeutic strategies to reduce the levels of prion proteins associated with prion diseases. By utilizing advanced editing techniques, the project aims to target and modify the genetic factors that contribute to the accumulation of these proteins. Patients may benefit from innovative treatments that could potentially halt or reverse the progression of prion diseases, which are currently untreatable. The approach involves cutting-edge molecular biology techniques to ensure precision in targeting the problematic proteins.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments that significantly improve the quality of life for patients suffering from prion diseases.
How similar studies have gone: While prion diseases are challenging to treat, there have been promising advancements in similar therapeutic editing approaches in other neurodegenerative conditions, suggesting potential for success in this area.
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.