How levels of the normal prion protein change how fast prion disease moves
How Substrate Dosage Drives Prion Disease Kinetics
This project looks at whether lowering the normal prion protein can slow or change the speed of prion disease for people affected by prion disorders.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-11238466 on NIH RePORTER |
What this research studies
Researchers will use animal models (including mice) and patient-derived prion samples to see how different amounts of the normal prion protein (PrP) change the steps of prion disease, such as initial misfolding, replication, and nerve damage. They will test genetic models with lower or higher PrP and use prion-lowering drugs called antisense oligonucleotides (ASOs) to mimic potential treatments. The team will compare effects across different prion strains and species and include spontaneous (not experimentally inoculated) disease models. Findings aim to show how PrP dosage relates to survival and which parts of the disease process are most affected by lowering PrP.
Who could benefit from this research
Good fit: Ideal candidates for sample donation or future trials would be people diagnosed with sporadic, familial, or acquired prion disease or individuals willing to provide biological samples for research.
Not a fit: People without prion disease or those expecting an immediate treatment benefit should not expect direct personal benefit from this basic and preclinical research at this stage.
Why it matters
Potential benefit: If successful, this work could guide PrP-lowering therapies that slow disease progression or extend survival for people with prion disease.
How similar studies have performed: Prior animal studies and early preclinical work with PrP-lowering antisense oligonucleotides have shown promise, and this project builds on those findings while addressing new questions about timing, species, and strain differences.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Vallabh, Sonia Minikel — Broad Institute, INC.
- Study coordinator: Vallabh, Sonia Minikel
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 below.