New tiny drugs to keep the aorta strong in Marfan syndrome
Official title: iPSC-derived VSMC for high-throughput screening of small molecules for aortic aneurysm
Funded at Boston University Medical Campus · NIH-11337519 · Data updated 2026-11-12
A small study tests new compounds that could keep the aorta from dilating and prevent serious complications in people with Marfan syndrome.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston University Medical Campus NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | June 1, 2025 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $122,625 |
| NIH project number | 5R21HL173511-02 |
| Project ID | NIH-11337519 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people with Marfan syndrome or other genetic factors predisposing to aortic aneurysm who could participate in future trials or provide samples.
✕Less likely to help
Patients without Marfan syndrome or related aortic conditions may not directly benefit from this specific study.
What this research studies
This project uses stem-cell derived cells that mimic the aorta to screen thousands of compounds for their ability to activate a protective enzyme called Sirt1, even when stressed by oxidation. The work combines computer design and lab testing to pick promising molecules that might stop destructive changes in the aorta. If successful, these molecules could become the basis for future therapies to prevent aneurysm and dissection in patients with Marfan-related aortic disease.
Why it matters
Potential benefit: If successful, the approach could lead to new medicines that reduce the risk of aortic aneurysm and life-threatening dissection for people with Marfan syndrome.
How similar studies have gone: Preliminary results with similar Sirt1-targeting strategies have shown potential in reducing damaging enzymes in vascular cells, but this exact approach is in early stages.
Where this research is happening
-
Boston University Medical Campus
Boston, United States Active
Researchers
- Principal investigator: Seta, Francesca — Boston University Medical Campus
- Study coordinator: Seta, Francesca
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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.