Understanding how Uba1 affects bone marrow health and inflammation
Defining the role of Uba1 in bone marrow physiology and inflammation
This study is looking into VEXAS syndrome, a serious condition that affects your immune system and blood cells, to understand how changes in a specific gene cause inflammation and other problems, which could help find better treatments for patients like you.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | New York University School of Medicine NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10998004 on NIH RePORTER |
What this research studies
This research investigates VEXAS syndrome, a severe autoinflammatory disorder linked to mutations in the UBA1 gene, which affects blood and immune cells. By using newly developed mouse models, the study aims to explore how these mutations lead to inflammation and disease progression. The research focuses on the role of UBA1 in bone marrow physiology, particularly how its loss impacts blood cell development and function. Patients may benefit from insights gained into the disease mechanisms, potentially leading to new treatment strategies.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with VEXAS syndrome or those exhibiting symptoms related to this condition.
Not a fit: Patients with conditions unrelated to UBA1 mutations or those not affected by autoinflammatory disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to better understanding and treatment options for patients with VEXAS syndrome.
How similar studies have performed: While there have been efforts to study VEXAS syndrome, this specific approach using newly established mouse models is relatively novel and untested.
Where this research is happening
New York, United States
- New York University School of Medicine — New York, United States (Active)
Researchers
- Principal investigator: Sawai-Frantz, Ayana — New York University School of Medicine
- Study coordinator: Sawai-Frantz, Ayana
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.