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-11145834 · Data updated 2026-11-12
This research aims to make gene therapy better at helping children with Duchenne muscular dystrophy breathe more easily.
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 | 2025 |
| Award (this year) | $648,119 |
| NIH project number | 5R01HL171282-02 |
| Project ID | NIH-11145834 on NIH RePORTER |
Who could benefit from this research
✓Could help
Future patients who could benefit from this research are those with Duchenne muscular dystrophy, particularly those experiencing or at risk of respiratory muscle weakness.
✕Less likely to help
Patients without Duchenne muscular dystrophy or those whose primary health concerns are unrelated to muscle weakness would not directly benefit from this specific gene therapy approach.
What this research studies
Duchenne muscular dystrophy is a serious condition that weakens muscles, including those needed for breathing, often leading to life-threatening respiratory failure. This project is working to understand how Duchenne affects both breathing muscles and the brain's control over breathing. We are using special mouse models that mimic Duchenne to test new ways to deliver gene therapy. The goal is to ensure that gene therapy can effectively reach and strengthen the breathing muscles, offering a potential new treatment for this critical aspect of the disease.
Why it matters
Potential benefit: If successful, this work could lead to more effective gene therapies that prevent or reverse the severe breathing difficulties experienced by patients with Duchenne muscular dystrophy.
How similar studies have gone: Previous preclinical gene therapy efforts have shown promising results in improving muscle function and survival, though more work is needed to specifically address respiratory outcomes.
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
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.
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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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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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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.