Two-gene AAV therapy for Limb-Girdle Muscular Dystrophy R9
Official title: (Project 1) Bicistronic Gene Therapies for the Muscular Dystrophies
Funded at Research Inst Nationwide Children's Hosp · NIH-11169982 · Data updated 2026-11-12
Developing a two-gene AAV gene therapy to replace the faulty gene and rebuild muscle for people with LGMDR9 (Limb Girdle Muscular Dystrophy Type R9).
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 | 2025 |
| Award (this year) | $294,439 |
| NIH project number | 5P50HD117373-02 |
| Project ID | NIH-11169982 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with genetically confirmed LGMDR9 (FKRP-related disease) who have ongoing muscle weakness but still retain enough muscle tissue for gene delivery would be the likely candidates.
✕Less likely to help
People with other forms of muscular dystrophy, or those with very advanced muscle loss where little muscle remains, are unlikely to benefit from this approach.
What this research studies
This project is finishing lab and safety work needed before asking the FDA to allow first human testing of a two-gene AAV therapy for LGMDR9. Researchers will compare the new bicistronic (two-gene) vector to single-gene vectors in preclinical models to see which best restores muscle size and strength. They will also test different formulations and perform safety studies required for a pre-IND filing. The goal is to show the approach can both prevent further muscle loss and promote muscle rebuilding before moving toward clinical trials.
Why it matters
Potential benefit: If successful, the therapy could both stop further muscle loss and help rebuild muscle size and strength in people with LGMDR9.
How similar studies have gone: AAV gene therapies have shown benefit in some neuromuscular diseases, but using a single AAV to deliver two genes for both replacement and muscle rebuilding is a new strategy with limited prior human testing.
Where this research is happening
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Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Martin, Paul Taylor — Research Inst Nationwide Children's Hosp
- Study coordinator: Martin, Paul Taylor
Related research
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Bicistronic gene therapy for limb girdle muscular dystrophy type R9
A patient-facing overview of a first-in-class gene therapy approach using two genes delivered by AAV to help people with LGMDR9.
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Understanding why scarring happens in muscular dystrophy muscles
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Improving Gene Therapies for Muscular Dystrophies
This center is working to improve gene therapies for Duchenne and Limb Girdle Muscular Dystrophy, aiming to make them safer and more effective for patients.
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Stopping Muscle Scarring in Muscular Dystrophies
This project explores how muscle scarring, called fibrosis, develops in muscular dystrophies like Duchenne MD, hoping to find new ways to help muscles heal better.
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.