Correcting mutations in the gene that causes cystic fibrosis
Gene Editing Strategies to Correct CFTR Mutations
This study is exploring a new way to fix specific gene mutations that cause cystic fibrosis, aiming to help improve lung health for people with this condition by using advanced gene editing techniques.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project ID | NIH-10892138 on NIH RePORTER |
What this research studies
This research focuses on developing gene editing techniques to correct mutations in the CFTR gene responsible for cystic fibrosis (CF). The approach utilizes advanced adenine base editing technology to target specific mutations that do not respond to existing therapies. By modifying the genetic code in somatic cells, the research aims to restore normal function to the CFTR protein, which is crucial for lung health. The study will specifically address several common mutations that lead to severe respiratory issues in CF patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with specific CFTR mutations that are not responsive to current treatments.
Not a fit: Patients with cystic fibrosis who have mutations that can be effectively treated with existing small molecule therapies may not 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 promising results using similar gene editing approaches, indicating potential for success in this area.
Where this research is happening
Iowa City, United States
- University of Iowa — Iowa City, United States (Active)
Researchers
- Principal investigator: Mccray, Paul B — University of Iowa
- Study coordinator: Mccray, Paul B
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.