Investigating how CFTR gene delivery affects lung function in cystic fibrosis
Role of disrupted ASL pH regulation in small airways in CF lung disease pathogenesis
This study is looking at how a gene called CFTR affects lung health in people with cystic fibrosis by using specially designed pigs to see if giving the gene back can help improve breathing.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Michigan State University NIH-funded |
| Lab location | 1 site (East Lansing, United States) |
| Project ID | NIH-11119733 on NIH RePORTER |
What this research studies
This research focuses on understanding the role of a specific gene, CFTR, in the development of lung disease in cystic fibrosis (CF) patients. Using a specially engineered pig model that mimics human CF, the study aims to explore how the absence of CFTR affects the small airways in the lungs. Researchers will deliver a copy of the CFTR gene using a viral vector to see if it can improve lung function by restoring the normal properties of airway surface liquid. This approach could provide insights into new treatments for CF lung disease.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those experiencing lung function decline.
Not a fit: Patients without cystic fibrosis or those with advanced lung disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that improve lung function and overall health for cystic fibrosis patients.
How similar studies have performed: While the approach of using gene therapy for cystic fibrosis is being explored, this specific method using the engineered pig model and AAV4 delivery is relatively novel.
Where this research is happening
East Lansing, United States
- Michigan State University — East Lansing, United States (Active)
Researchers
- Principal investigator: Li, Xiaopeng — Michigan State University
- Study coordinator: Li, Xiaopeng
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.