Investigating biomarkers related to prion disease and its treatment
Biomarkers and Mechanisms of PrP Misfolding, Mutation, and Deficiency
This study is looking for signs in blood and spinal fluid that can help us understand how prion disease progresses and responds to treatments, so we can find better ways to detect and treat this serious condition in people at risk.
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-11057683 on NIH RePORTER |
What this research studies
This research focuses on understanding prion disease, a fatal neurodegenerative condition, by identifying molecular biomarkers that indicate the disease's progression and response to treatment. The study will analyze cerebrospinal fluid and plasma samples from healthy individuals at risk and from infected animal models to track changes in prion protein (PrP) misfolding. By discovering these biomarkers, the research aims to improve early detection and treatment strategies for prion disease, potentially leading to better patient outcomes. The findings could help determine the safety and effectiveness of new therapies as they advance toward clinical use.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with a genetic predisposition to prion disease and those in the early stages of the disease.
Not a fit: Patients with advanced prion disease or those without any genetic risk factors for the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to earlier diagnosis and more effective treatments for prion disease, improving survival and quality of life for affected patients.
How similar studies have performed: Previous research has shown promise in using biomarkers for other neurodegenerative diseases, suggesting that this approach could be effective for prion disease as well.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Minikel, Eric Vallabh — Broad Institute, INC.
- Study coordinator: Minikel, Eric Vallabh
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.