Gene therapy for cystic fibrosis using viral vectors
Official title: Cystic Fibrosis Gene Therapy with adeno-associated viral vectors
Funded at Johns Hopkins University · NIH-11125754 · Data updated 2026-11-12
This study is exploring a new way to use gene therapy to help people with cystic fibrosis by improving a delivery method that targets the lungs, and it's testing this approach in animals to make sure it's safe and effective for all CF patients, no matter their specific gene changes.
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 start | July 1, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $730,685 |
| NIH project number | 5R01HL167797-02 |
| Project ID | NIH-11125754 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with various mutations in the CF gene.
✕Less likely to help
Patients with conditions unrelated to cystic fibrosis or those who do not have a CF diagnosis may not benefit from this research.
What this research studies
This research investigates the use of adeno-associated viral (AAV) vectors to deliver gene therapy for cystic fibrosis (CF). The approach focuses on developing new strategies to enhance the effectiveness of AAV vectors, particularly targeting the lung and addressing various mutations in the CF gene. By utilizing animal models, including Rhesus macaques and ferrets, the study aims to evaluate the safety and efficacy of these gene therapies. The goal is to create a treatment that can benefit all CF patients, regardless of their specific genetic mutations.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking gene therapy option for all cystic fibrosis patients, potentially improving their lung function and overall health.
How similar studies have gone: Previous studies have shown promise in using AAV vectors for gene therapy, but this approach aims to address limitations that have hindered consistent therapeutic effects.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Cebotaru, Liudmila — Johns Hopkins University
- Study coordinator: Cebotaru, Liudmila
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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.