MDSRC for Next Directions in Muscular Dystrophy Research
Funded at Research Inst Nationwide Children's Hosp · NIH-11397440 · Data updated 2026-11-12
A major research center grant aims to speed new gene therapy ideas for muscular dystrophy by sharing expertise, creating tools, and advancing three focused projects.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | August 15, 2024 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,487,548 |
| NIH project number | 5P50HD117373-03 |
| Project ID | NIH-11397440 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with muscular dystrophy, especially LGMD2I, could benefit from advances in gene therapy approaches and biomarker development.
✕Less likely to help
Patients without muscular dystrophy or without access to participating centers may not directly benefit from these specific projects.
What this research studies
This grant supports the Center for Gene Therapy at Nationwide Children’s Hospital to help muscular dystrophy researchers access core resources and collaborate. It funds three projects: a bicistronic AAV vector to prevent disease and improve muscle function in LGMD2I; vector designs using U7snRNAs to restore dystrophin; and discovery of new biomarkers while improving AAV delivery and reducing immune responses. The effort emphasizes translating lab findings to patient care and building a shared resource for the broader community. The Muscular Dystrophy Snyder Resource Center (MDSRC) will leverage cores to accelerate these translational efforts.
Why it matters
Potential benefit: If successful, patients with muscular dystrophy could see faster development of gene therapies and better tools for diagnosis and monitoring.
How similar studies have gone: Prior research shows promise in gene therapy vectors and biomarker discovery, but this center aims to accelerate translation through shared resources.
Where this research is happening
-
Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Flanigan, Kevin M — Research Inst Nationwide Children's Hosp
- Study coordinator: Flanigan, Kevin M
Related research
Other NIH-funded projects on the same topics.
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Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
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Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
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Endocrine role of intramuscular fat in muscular dystrophy
This grant studies how fat that builds up inside muscles in Duchenne and Becker muscular dystrophy may release hormones that affect overall health, especially metabolism.
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Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
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Curation of muscular dystrophy genes to improve diagnosis and care
A grant to help doctors better interpret genetic tests for muscular dystrophy and myopathy so patients get clearer diagnoses and faster access to therapies.
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MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
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.