AAV gene therapy to fix the CFTR defect in cystic fibrosis
Cystic Fibrosis Gene Therapy with adeno-associated viral vectors
A lung-targeted AAV gene therapy aims to deliver a corrected CFTR gene to help people with cystic fibrosis, including those whose mutations are not helped by existing drugs.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project ID | NIH-11312654 on NIH RePORTER |
What this research studies
Researchers plan to use a lung-tropic AAV1 viral vector carrying a shortened CFTR gene and a strong promoter to improve delivery to airway cells. They will first study safety and how the therapy behaves in the body using rhesus macaques and ferrets that carry the F508 CF mutation. The work builds on earlier AAV safety data and on recent advances in CFTR-correcting drugs, with the goal of a gene replacement approach that could work across many CF mutations. If these preclinical studies show acceptable safety and promising activity, the team would use that data to support future human trials.
Who could benefit from this research
Good fit: People with cystic fibrosis of various CFTR mutations — particularly those not helped by current modulators — would be the intended candidates for eventual human testing.
Not a fit: Those with advanced, irreversible lung damage or individuals with strong pre-existing immunity to the AAV vector that blocks delivery may not benefit from this approach.
Why it matters
Potential benefit: If successful, the approach could restore CFTR function across many mutations and substantially reduce lung infections and breathing problems for people with CF.
How similar studies have performed: Previous AAV-based CF trials have shown safety but not consistent clinical benefit, so this program uses newer vector choices and a shortened CFTR to try to improve on past results.
Where this research is happening
Baltimore, United States
- Johns Hopkins University — Baltimore, United States (Active)
Researchers
- Principal investigator: Cebotaru, Liudmila — Johns Hopkins University
- Study coordinator: Cebotaru, Liudmila
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.