Using mineralocorticoid receptor blockers to reduce inflammation in Duchenne muscular dystrophy
Official title: Mechanisms of mineralocorticoid receptor antagonism on inflammation in muscular dystrophy
Funded at Ohio State University · NIH-11251255 · Data updated 2026-11-12
This project sees if drugs that block mineralocorticoid receptors can lower muscle inflammation and help AAV gene therapy work better for people with Duchenne muscular dystrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ohio State University NIH-funded |
| Lab location | 1 site (Columbus, UNITED STATES) |
| Project start | January 1, 2022 |
| Project end | December 31, 2026 |
| Funding year | 2026 |
| Award (this year) | $416,861 |
| NIH project number | 5R01NS124681-05 |
| Project ID | NIH-11251255 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with Duchenne muscular dystrophy, especially those receiving or considering AAV-based micro-dystrophin gene therapy, are the most relevant candidates.
✕Less likely to help
People with other types of muscle disease or those who are not eligible for AAV gene therapy are unlikely to benefit directly from this work.
What this research studies
This project studies whether adding medicines that block the mineralocorticoid receptor can reduce the chronic inflammation that follows AAV micro-dystrophin gene therapy. Researchers will use laboratory models of Duchenne muscular dystrophy and tissue analyses to measure muscle inflammation, membrane stability, fibrosis, and strength. They will examine effects on muscle stem cell repair and whether the drugs help AAV-delivered micro-dystrophin remain effective for longer. The goal is to identify a drug combination that could be translated into safer, longer‑lasting treatments for people with DMD.
Why it matters
Potential benefit: If successful, this approach could lower muscle and heart inflammation, protect muscle cells from damage, and extend the benefits of AAV gene therapy.
How similar studies have gone: Mineralocorticoid receptor blockers have shown clinical benefit for DMD-related heart disease and positive effects on muscle in preclinical studies, but combining them with AAV gene therapy is a novel strategy not yet proven in patients.
Where this research is happening
-
Ohio State University
Columbus, United States Active
Researchers
- Principal investigator: Rafael-Fortney, Jill a — Ohio State University
- Study coordinator: Rafael-Fortney, Jill a
Related research
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Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
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This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
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.