Improving Gene Therapy for Breathing Problems in Duchenne Muscular Dystrophy
Optimizing Gene Therapy for Respiratory Insufficiency in Duchenne Muscular Dystrophy
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 ID | NIH-11145834 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Future patients who could benefit from this research are those with Duchenne muscular dystrophy, particularly those experiencing or at risk of respiratory muscle weakness.
Not a fit: 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.
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 performed: 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
Durham, United States
- Duke University — Durham, United States (Active)
Researchers
- Principal investigator: Elmallah, Mai — Duke University
- Study coordinator: Elmallah, Mai
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 below.