Developing a gene therapy to lower prion protein levels for treating prion disease
Official title: Research Core (Deverman)
Funded at Broad Institute, INC. · NIH-11085946 · Data updated 2026-11-12
This study is testing a new gene therapy that could help people with prion diseases by lowering the harmful protein that causes these conditions, potentially offering a one-time treatment that might slow down or prevent the disease from getting worse.
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) | $1,520,416 |
| NIH project number | 5U19NS132315-03 |
| Project ID | NIH-11085946 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults over 21 years old who are at risk of or currently affected by prion diseases.
✕Less likely to help
Patients with prion diseases who are under 21 years old or those with other unrelated neurodegenerative conditions may not benefit from this research.
What this research studies
This research focuses on creating a gene therapy that aims to permanently reduce levels of the prion protein (PrP), which is responsible for fatal neurodegenerative diseases. The approach involves a multi-disciplinary team utilizing advanced techniques such as base editing and vector engineering to enhance gene delivery to the central nervous system. By targeting the underlying cause of prion diseases, the research seeks to provide a long-lasting treatment option for affected individuals. Patients may benefit from a single-dose therapy that could prevent or delay the progression of these diseases.
Why it matters
Potential benefit: If successful, this research could offer a groundbreaking treatment option for patients suffering from prion diseases, potentially improving their quality of life and extending survival.
How similar studies have gone: Other research has shown promise in using gene therapy approaches for neurodegenerative diseases, indicating potential success for this novel application.
Where this research is happening
-
Broad Institute, INC.
Cambridge, United States Active
Researchers
- Principal investigator: Deverman, Benjamin E — Broad Institute, INC.
- Study coordinator: Deverman, Benjamin E
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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.