Correcting genetic mutations in cystic fibrosis airways
Adenine Base Edited Correction of Cystic Fibrosis Airways
This study is exploring a new way to fix the genetic problems that cause cystic fibrosis by using a special tool to help your body make the proteins it needs for healthy lungs, and it's testing two different ways to deliver this tool to the cells in your airways.
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-11003727 on NIH RePORTER |
What this research studies
This research focuses on developing a new method to correct genetic mutations that cause cystic fibrosis (CF) by using a specialized tool called an Adenine Base Editor (ABE). The ABE works by converting specific genetic material to produce the necessary proteins for lung function, particularly in patients whose CFTR proteins are only partially produced. The researchers aim to effectively deliver this editing tool to airway cells using two different delivery methods: adeno-associated viruses (AAVs) and viral-like particles (VLPs). By comparing these methods, the study seeks to find the most effective way to treat CF at the genetic level.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with cystic fibrosis who have specific genetic mutations that prevent the production of functional CFTR proteins.
Not a fit: Patients with cystic fibrosis who do not have the targeted genetic mutations or those who are already benefiting from existing therapies may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for cystic fibrosis patients who currently have no effective therapies available.
How similar studies have performed: Other research has shown promise in using gene editing techniques for similar genetic conditions, indicating that this approach could lead to significant advancements in treatment.
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.