Understanding and treating congenital aortic valve problems
Mechanisms and Therapeutics for Congenital Aortic Valve Disease
This project develops medicines targeting the genetic and molecular causes of malformed aortic valves to help people born with bicuspid or other congenital valve problems avoid or delay surgery.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project ID | NIH-11310034 on NIH RePORTER |
What this research studies
From your point of view, the team looks at inherited gene changes linked to bicuspid and malformed aortic valves and how those changes alter valve cells. They follow specific pathways (including NOTCH1 and nitric oxide signaling) and study the role of GATA5 using animal models and human valve tissue. Lab experiments will be used to find molecular targets and test candidate drugs that might slow valve narrowing. Promising lab findings would be pushed toward steps needed for future clinical testing in patients.
Who could benefit from this research
Good fit: People born with bicuspid aortic valve or other forms of congenital aortic valve stenosis who are at risk of progressive valve narrowing would be the most relevant candidates.
Not a fit: Patients whose valve narrowing is caused mainly by age-related calcific aortic stenosis or who already need immediate valve replacement are unlikely to benefit from these early-stage therapies.
Why it matters
Potential benefit: If successful, this work could produce medicines that slow or prevent progression of congenital aortic valve stenosis and reduce the need for valve surgery or replacement.
How similar studies have performed: Previous genetic and animal studies have established key roles for NOTCH1, GATA5, and nitric oxide pathways, but effective pharmacologic treatments for congenital aortic valve stenosis remain largely unproven.
Where this research is happening
Columbus, United States
- Research Inst Nationwide Children's Hosp — Columbus, United States (Active)
Researchers
- Principal investigator: Garg, Vidu — Research Inst Nationwide Children's Hosp
- Study coordinator: Garg, Vidu
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.