Improving gene therapy for breathing problems in Duchenne muscular dystrophy
Official title: Optimizing Gene Therapy for Respiratory Insufficiency in Duchenne Muscular Dystrophy
Funded at Duke University · NIH-10980431 · Data updated 2026-11-12
This study is looking at a new gene therapy to help improve breathing and muscle health for people with Duchenne muscular dystrophy (DMD) by using a special virus to deliver a helpful gene, and it's being tested in mouse models that mimic human conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | July 29, 2024 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $610,819 |
| NIH project number | 1R01HL171282-01A1 |
| Project ID | NIH-10980431 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are young children diagnosed with Duchenne muscular dystrophy, particularly those experiencing respiratory insufficiency.
✕Less likely to help
Patients with other forms of muscular dystrophy or those who are not experiencing respiratory issues may not benefit from this research.
What this research studies
This research focuses on Duchenne muscular dystrophy (DMD), a severe genetic disorder that leads to muscle degeneration and respiratory failure. The team aims to optimize gene therapy using adeno-associated viruses (AAV) to deliver a microdystrophin gene, which is crucial for muscle function, particularly in respiratory muscles. By studying humanized mouse models, the researchers will assess how well this therapy can improve breathing and overall muscle health, potentially leading to better treatment options for patients with DMD.
Why it matters
Potential benefit: If successful, this research could significantly enhance the quality of life and survival rates for patients with Duchenne muscular dystrophy by improving respiratory function.
How similar studies have gone: Previous studies using AAV-based gene therapies have shown promising results in improving muscle function, indicating potential success for this approach in treating DMD.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Elmallah, Mai — Duke University
- Study coordinator: Elmallah, Mai
Related research
Other NIH-funded projects on the same topics.
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Optimizing gene therapy to help breathing in Duchenne muscular dystrophy
A Duke University team is testing a gene therapy approach to improve breathing in boys with Duchenne muscular dystrophy.
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Improving Gene Therapy for Breathing Problems in Duchenne Muscular Dystrophy
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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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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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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.