Improving airway health for cystic fibrosis patients using corrected stem cells
Optimizing Surgical Transplant of CFTR Gene-Corrected Human Basal Stem Cells to the Upper Airway
This study is testing a new way to help people with cystic fibrosis by using special stem cells that have been corrected to fix a common gene problem, with the hope of improving breathing and reducing infections.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-11010390 on NIH RePORTER |
What this research studies
This research focuses on developing a new treatment for cystic fibrosis (CF) by using gene-corrected human airway basal stem cells. The approach involves correcting the common CFTR gene mutation in these stem cells using advanced CRISPR technology, which is then transplanted into the upper airway of CF patients. By restoring the function of these cells, the research aims to improve airway function and reduce the frequency of infections and inflammation associated with CF. The study is designed to provide a novel, durable therapy for patients who currently have limited treatment options.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with the ∆F508 mutation.
Not a fit: Patients without cystic fibrosis or those who do not have the ∆F508 mutation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to significant improvements in respiratory health and quality of life for cystic fibrosis patients.
How similar studies have performed: Other research has shown promising results using similar gene-editing approaches in stem cell therapies, indicating potential for success in this novel application.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Nayak, Jayakar V — Stanford University
- Study coordinator: Nayak, Jayakar V
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.