Oxidative changes and aortic aneurysms in Marfan syndrome
Thiol redox signaling in aortic aneurysm
['FUNDING_R01'] · BOSTON UNIVERSITY MEDICAL CAMPUS · NIH-11328829
This work looks at whether reversing a chemical modification that turns off the enzyme Sirt1 in aortic wall cells can help prevent dangerous aortic enlargement in people with Marfan syndrome.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BOSTON UNIVERSITY MEDICAL CAMPUS (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11328829 on ClinicalTrials.gov |
What this research studies
From a patient's viewpoint, researchers compare aortic tissue from people with Marfan syndrome and a mouse model to find chemical changes that inactivate Sirt1 in the aorta. They will study how glutathionylation (an oxidative modification) affects vascular smooth muscle cells and promotes aneurysm formation. The team plans to use molecular tools, including gene delivery methods, to restore Sirt1 activity in vessel cells and watch whether that slows or prevents aortic wall degeneration. Results will guide whether targeting this redox pathway could become a therapy to protect the aorta.
Who could benefit from this research
Good fit: People diagnosed with Marfan syndrome (FBN1 mutation) who have or are at risk for thoracic aortic aneurysm would be the most relevant candidates for related future therapies or tissue donation.
Not a fit: People whose aneurysms are caused by unrelated conditions or who already need urgent surgical repair are unlikely to benefit directly from this molecular work.
Why it matters
Potential benefit: If successful, this could lead to new treatments that slow or prevent aortic enlargement and lower the risk of dissection in people with Marfan syndrome.
How similar studies have performed: Previous work has shown increased oxidative stress in aneurysms and protective roles for Sirt1, but directly targeting Sirt1 glutathionylation as a therapy is a newer, less-tested approach.
Where this research is happening
BOSTON, UNITED STATES
- BOSTON UNIVERSITY MEDICAL CAMPUS — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SETA, FRANCESCA — BOSTON UNIVERSITY MEDICAL CAMPUS
- Study coordinator: SETA, FRANCESCA
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.