Understanding lung injury and healing in Pompe Disease
Official title: Alveolar injury and repair in Pompe Disease
Funded at Duke University · NIH-11041606 · Data updated 2026-11-12
This study is looking at how Pompe Disease affects lung cells and their healing abilities, especially focusing on a part of the cells that helps keep your lungs working well, to find out why people with this condition often have breathing problems, and it’s exploring a way to deliver the missing enzyme to help improve lung health.
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 | January 29, 2025 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $762,974 |
| NIH project number | 1R01HL177642-01 |
| Project ID | NIH-11041606 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Pompe Disease, particularly those experiencing respiratory complications.
✕Less likely to help
Patients with Pompe Disease who do not have respiratory issues may not benefit directly from this research.
What this research studies
This research investigates how Pompe Disease, caused by a deficiency in the enzyme acid alpha-glucosidase, affects lung cells and their ability to repair after injury. The study focuses on the role of lysosomes in alveolar cells, which are crucial for maintaining lung function. By examining the cellular architecture and autophagy processes in these cells, the research aims to uncover why patients with Pompe Disease often experience respiratory issues despite existing treatments. The approach includes using adeno-associated viruses to deliver the missing enzyme to improve lung health.
Why it matters
Potential benefit: If successful, this research could lead to improved treatments that enhance lung function and reduce respiratory complications in patients with Pompe Disease.
How similar studies have gone: Previous research has shown promising results in addressing similar respiratory complications in other genetic disorders, indicating potential for success in this area.
Where this research is happening
-
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.