Understanding how a specific protein pathway affects inflammation in a rare disease.
Regulation of inflammation by the ubiquitin proteasome pathway
['FUNDING_R01'] · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · NIH-11015878
This study is looking into VEXAS syndrome, a rare condition that causes inflammation and blood issues, to better understand how it works and find new treatments that could help people living with it.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NEW YORK UNIVERSITY SCHOOL OF MEDICINE (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11015878 on ClinicalTrials.gov |
What this research studies
This research focuses on VEXAS syndrome, a newly identified inflammatory and hematologic disease caused by mutations in the UBA1 gene. The study aims to explore the mechanisms behind this condition, which presents symptoms similar to other rheumatic diseases but with unique challenges, including high mortality and resistance to treatment. By investigating the role of the ubiquitin proteasome pathway in inflammation, researchers hope to uncover new insights that could lead to effective therapies for affected individuals. The approach includes studying patient-derived cells and cellular models to identify activated inflammatory pathways and their implications for treatment.
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 UBA1 mutations.
Not a fit: Patients without VEXAS syndrome or those not exhibiting symptoms related to UBA1 mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of targeted therapies for patients suffering from VEXAS syndrome.
How similar studies have performed: While VEXAS syndrome is a newly identified condition, similar research approaches have shown promise in understanding other inflammatory diseases, suggesting potential for success in this area.
Where this research is happening
NEW YORK, UNITED STATES
- NEW YORK UNIVERSITY SCHOOL OF MEDICINE — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BECK, DAVID — NEW YORK UNIVERSITY SCHOOL OF MEDICINE
- Study coordinator: BECK, DAVID
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.