Understanding the CFTR Protein in Cystic Fibrosis
The CFTR Interactome
This research aims to better understand the faulty CFTR protein that causes Cystic Fibrosis, especially for patients whose condition doesn't improve with current medications.
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-11136978 on NIH RePORTER |
What this research studies
Cystic Fibrosis is a common inherited childhood disease caused by a faulty CFTR gene, and while some patients benefit from existing drugs, many experience side effects or don't respond to treatment at all. This project is developing a new, more sensitive method called bioTMT-CPP to precisely map the structural problems in the faulty CFTR protein. By understanding these defects at a detailed level, we hope to pave the way for new and more effective treatments for all patients with Cystic Fibrosis.
Who could benefit from this research
Good fit: This foundational research is relevant to all patients with Cystic Fibrosis, particularly those with mutations that do not respond well to existing therapies.
Not a fit: Patients whose CFTR mutations are already effectively managed by current drug combinations may not see direct immediate benefit from this basic science work, though it could lead to future improvements.
Why it matters
Potential benefit: If successful, this work could lead to the development of new, more effective drugs with fewer side effects for patients with Cystic Fibrosis, especially those who currently have limited treatment options.
How similar studies have performed: This project builds upon a previously developed method called Covalent Protein Painting (CPP), indicating a foundation of prior success in similar structural characterization approaches.
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.