Developing a new therapy to lower prion proteins for treating prion disease
Official title: Advancement of Prion Protein-Lowering Divalent siRNA Therapy for Prion Disease
Funded at Broad Institute, INC. · NIH-10897190 · Data updated 2026-11-12
This study is looking at a new treatment that uses special technology to help reduce harmful proteins in the brain that cause prion diseases, like Creutzfeldt-Jakob disease, and it's being tested in animals first to see if it works safely before moving on to people.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | August 1, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $806,536 |
| NIH project number | 5U01NS132994-02 |
| Project ID | NIH-10897190 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with prion diseases such as Creutzfeldt-Jakob disease or those at risk due to genetic factors.
✕Less likely to help
Patients with prion diseases who are already in advanced stages of the condition may not benefit from this research.
What this research studies
This research focuses on prion diseases, which are fatal neurodegenerative conditions caused by misfolded prion proteins in the brain. The study aims to develop a novel therapy using divalent siRNA technology to effectively reduce the levels of these harmful proteins. By targeting the RNA that encodes the prion protein, the therapy seeks to prevent or slow the progression of diseases like Creutzfeldt-Jakob disease. The approach involves testing the effectiveness and safety of this new treatment in animal models before considering human applications.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking therapy that significantly slows down or prevents the progression of prion diseases.
How similar studies have gone: Previous research has shown promise in using RNA-based therapies for neurodegenerative diseases, indicating potential success for this novel approach.
Where this research is happening
-
Broad Institute, INC.
Cambridge, United States Active
Researchers
- Principal investigator: Minikel, Eric Vallabh — Broad Institute, INC.
- Study coordinator: Minikel, Eric Vallabh
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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.