Adenine base editing to repair CF airway cells
Adenine Base Edited Correction of Cystic Fibrosis Airways
This project uses a gentle gene-editing tool called an adenine base editor delivered by viral or virus-like carriers to restore CFTR protein in people with cystic fibrosis who have mutations that prevent full protein production.
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-11257705 on NIH RePORTER |
What this research studies
Researchers are using adenine base editors (a form of CRISPR that changes a single DNA letter without cutting both DNA strands) to correct mutations that stop CFTR protein production. They previously fixed nonsense mutations in human airway cells in the lab and will now compare two delivery methods — adeno-associated viruses (AAVs) and virus-like particles (VLPs) — to see which gets the editor into enough of the right airway cells. Because the lung has a huge surface area and barriers to uptake, the team will test delivery efficiency in models that mimic human airways before moving toward studies needed for future patient treatments. The goal is to overcome the delivery hurdle that has kept gene editing from reaching many parts of the lung.
Who could benefit from this research
Good fit: People with cystic fibrosis who carry nonsense or other mutations that produce little or no CFTR protein and who meet the study's health and age criteria would be the ideal candidates.
Not a fit: People whose CFTR mutations are already effectively treated with current modulator drugs or who do not carry the targeted mutation types are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this could restore functional CFTR in people with mutations not helped by existing drugs and reduce lung infections and breathing problems.
How similar studies have performed: Base-editing has corrected CFTR mutations in lab-grown human airway cells, and gene-delivery approaches have shown promise, but widespread safe delivery across the lung remains largely unproven.
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.