Understanding the CFTR protein and its role in cystic fibrosis
The CFTR Interactome
This study is looking at how different changes in a gene called CFTR cause cystic fibrosis in kids, and it aims to find new ways to help those who don’t get better with current treatments by exploring the structure of the faulty proteins.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scripps Research Institute, the NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-10889243 on NIH RePORTER |
What this research studies
This research focuses on cystic fibrosis (CF), a genetic condition caused by mutations in the CFTR gene, which affects many children. The project aims to improve the understanding of how different mutations in the CFTR protein lead to disease symptoms and to develop new therapies for patients who do not respond to existing treatments. By using a novel method called bioTMT-CPP, researchers will analyze the structure of misfolded CFTR proteins to identify potential new drug targets. This approach could lead to more effective treatments with fewer side effects for children with CF.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with cystic fibrosis, particularly those with mutations in the CFTR gene that are currently difficult to treat.
Not a fit: Patients with cystic fibrosis who have mutations that are already effectively managed by existing therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the quality of life for children with cystic fibrosis.
How similar studies have performed: Previous research has shown promise in using structural analysis techniques to develop new therapies for genetic conditions, indicating that this approach could be successful.
Where this research is happening
La Jolla, United States
- Scripps Research Institute, the — La Jolla, United States (Active)
Researchers
- Principal investigator: Yates Iii, John R — Scripps Research Institute, the
- Study coordinator: Yates Iii, John R
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.