Therapeutic editing to lower PrP in prion disease
Funded at Broad Institute, INC. · NIH-11579210 · Data updated 2026-11-12
A research program aims to lower the prion protein (PrP) in people at risk of or living with prion disease using a one-time gene-editing approach.
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 | June 25, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $935,981 |
| NIH project number | 3U19NS132315-04S1 |
| Project ID | NIH-11579210 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include individuals with prion disease or those at risk due to genetic factors and who could be eligible for future gene-therapy trials.
✕Less likely to help
People without prion disease or without access to future gene-therapy trials may not directly benefit.
What this research studies
This program focuses on developing a safe, permanent way to reduce PrP levels in humans by using gene-editing and related delivery methods. It brings together experts in prion biology, genome editing, and viral vectors to test different editing strategies. The goal is to create a one-time treatment that can slow or prevent prion neurodegeneration across disease forms and stages. The work involves preclinical development with a focus on translating advances into a potential human therapy.
Why it matters
Potential benefit: If successful, this approach could provide a long-lasting treatment that lowers disease-causing PrP and slows progression.
How similar studies have gone: Prior antisense oligonucleotide work and genetic proof-of-concept support the approach, with ongoing exploration of gene-editing strategies.
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.