Durable gene therapy for CF: delivering fixes to airway basal cells
Official title: Editing basal cells for durable gene therapy of cystic fibrosis
Funded at University of Texas at Austin · NIH-11394545 · Data updated 2026-11-12
Aims to develop a special inhaled gene-delivery system to reach airway stem cells in people with cystic fibrosis who have certain mutations, offering a potential lasting correction.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Texas at Austin NIH-funded |
| Lab location | 1 site (Austin, United States) |
| Project start | August 15, 2025 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $489,651 |
| NIH project number | 5R01HL177349-02 |
| Project ID | NIH-11394545 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with CF who have nonsense CFTR mutations and are eligible for inhaled gene therapy approaches.
✕Less likely to help
Patients without nonsense CFTR mutations or those unable to receive inhaled gene therapies may not benefit.
What this research studies
This project focuses on creating peptide-coated lipid nanoparticles that can be inhaled to reach the basal cells in the lungs. By targeting these stem-like cells, the therapy hopes to permanently correct the CFTR mutation rather than provide only temporary relief. The work involves designing delivery particles, testing their ability to traverse mucus, reach basal cells, and introduce a corrected CFTR gene in a way that could endure over time. It is centered on translating per-patient benefits through a more durable treatment option for CF lung disease.
Why it matters
Potential benefit: If successful, this approach could lead to a one-time or long-lasting correction of CFTR in the lungs, reducing disease progression for patients with nonsense CFTR mutations.
How similar studies have gone: Previous inhaled gene therapy efforts struggled to reach airway basal cells; this project aims to overcome that gap with targeted pepLNPs.
Where this research is happening
-
University of Texas at Austin
Austin, United States Active
Researchers
- Principal investigator: Ghosh, Debadyuti — University of Texas at Austin
- Study coordinator: Ghosh, Debadyuti
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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.