Investigating a new approach to treat sickle cell disease using biased signaling
Biased PAR1 Agonism in Sickle Cell Disease
This study is looking at how a specific receptor in the body can help people with sickle cell disease by finding better ways to use certain proteins to reduce pain and hospital visits.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project ID | NIH-11009949 on NIH RePORTER |
What this research studies
This research focuses on sickle cell disease (SCD), a condition caused by a mutation in the β-globin gene that leads to painful vaso-occlusive crises and chronic anemia. The study explores the role of a receptor called PAR1, which is activated by different proteins, including thrombin and activated protein C (APC). By comparing these activation pathways, the research aims to find a way to enhance the protective effects of APC while minimizing harmful effects associated with thrombin. This could lead to new treatment strategies that improve patient outcomes and reduce hospitalizations due to SCD.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with sickle cell disease, particularly those experiencing frequent vaso-occlusive crises.
Not a fit: Patients who do not have sickle cell disease or those with other unrelated health conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved treatments for sickle cell disease, reducing pain crises and hospital visits for patients.
How similar studies have performed: Previous studies have shown promise in targeting PAR1 signaling pathways, suggesting that this approach could lead to significant advancements in treating sickle cell disease.
Where this research is happening
Chapel Hill, United States
- Univ of North Carolina Chapel Hill — Chapel Hill, United States (Active)
Researchers
- Principal investigator: Sparkenbaugh, Erica M — Univ of North Carolina Chapel Hill
- Study coordinator: Sparkenbaugh, Erica M
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.