Improving Gene Therapies for Muscular Dystrophies
Official title: Addressing Shortcomings of AAV GeneTherapies for the Muscular Dystrophies
Funded at University of Florida · NIH-11130228 · Data updated 2026-11-12
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.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | September 25, 2005 |
| Project end | July 31, 2030 |
| Funding year | 2025 |
| Award (this year) | $1,347,536 |
| NIH project number | 9P50HD119693-20 |
| Project ID | NIH-11130228 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is relevant for patients with Duchenne Muscular Dystrophy and Limb Girdle Muscular Dystrophy, especially those who might be candidates for gene therapy.
✕Less likely to help
Patients without Duchenne or Limb Girdle Muscular Dystrophy would not directly benefit from this specific gene therapy approach.
What this research studies
This research center is dedicated to making gene therapies better for people with Duchenne and Limb Girdle Muscular Dystrophy. We are creating new versions of gene therapy that can be delivered more effectively to muscles and the heart. Our work also focuses on reducing unwanted immune responses to these therapies, which can be a challenge with current treatments. We are testing these new approaches in lab models and plan to move towards human studies, including a clinical study to follow treated boys.
Why it matters
Potential benefit: If successful, this work could lead to more effective and safer gene therapy options for individuals living with Duchenne and Limb Girdle Muscular Dystrophy.
How similar studies have gone: While AAV gene therapies are already being explored, this work aims to address known limitations and develop novel, improved approaches.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Sweeney, H Lee — University of Florida
- Study coordinator: Sweeney, H Lee
Related research
Other NIH-funded projects on the same topics.
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Noninvasive breathing imaging to guide care for people with muscular dystrophy
A noninvasive imaging tool called electrical impedance tomography (EIT) will be studied to help decide when to start noninvasive ventilation for people with muscular dystrophy.
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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
Researchers are studying why scar tissue forms in muscles affected by muscular dystrophy and how to slow or reverse it in patients.
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Addressing Shortcomings of AAV Gene Therapies for Muscular Dystrophy
A national research center is working to improve AAV-based gene therapies for Duchenne and related muscular dystrophies so that patients may get safer, more effective treatments.
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Two-gene AAV therapy for Limb-Girdle Muscular Dystrophy R9
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).
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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.