One-time inhaled gene therapy to fix cystic fibrosis airways
Life-long phenotypic correction of CF airways
This project is developing a single-dose inhaled gene therapy to restore the faulty CFTR protein in people with cystic fibrosis, including those who don't benefit from current drugs.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project ID | NIH-11287854 on NIH RePORTER |
What this research studies
Researchers plan to use specially engineered adenoviral vectors delivered by aerosol to reach airway progenitor cells so the correction could last a lifetime from one dose. They are comparing two Ad-based strategies, including chimeric Ad5 vectors with fibers from species B adenoviruses, to find which best targets the right cells and penetrates airway mucus. The team will address known problems with adenoviral therapy such as short-lived expression and immune reactions while testing safety and durability in lab and animal models. The work aims to move promising approaches toward future clinical testing for people with CF.
Who could benefit from this research
Good fit: Ideal candidates would be people with cystic fibrosis, especially those whose CFTR mutations do not respond to existing modulator drugs.
Not a fit: People without lung disease, those with severe irreversible lung damage, or those medically ineligible for gene therapy (for example due to immune issues) may not benefit from this approach.
Why it matters
Potential benefit: If successful, a single inhaled treatment could permanently restore lung CFTR function and greatly reduce lung disease in people with cystic fibrosis.
How similar studies have performed: Adenoviral gene delivery has been tried before with limited and transient benefit, so this work builds on prior experience but seeks new vector designs and delivery methods to achieve durable correction.
Where this research is happening
Iowa City, United States
- University of Iowa — Iowa City, United States (Active)
Researchers
- Principal investigator: Sinn, Patrick L — University of Iowa
- Study coordinator: Sinn, Patrick L
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.