Understanding how CFTR correctors help treat cystic fibrosis
Investigating of the Mechanisms of Action of CFTR Correctors in RescuingDelta F508-CFTR
['FUNDING_R01'] · CEDARS-SINAI MEDICAL CENTER · NIH-10656430
This study is looking at how certain medications can help improve lung function for people with cystic fibrosis who have a common mutation called ΔF508, by making more of the healthy CFTR protein available in their cells.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CEDARS-SINAI MEDICAL CENTER (nih funded) |
| Locations | 1 site (LOS ANGELES, UNITED STATES) |
| Trial ID | NIH-10656430 on ClinicalTrials.gov |
What this research studies
This research investigates how specific drugs, known as CFTR correctors, can improve the function of a common mutation in cystic fibrosis called ΔF508. The study aims to understand the mechanisms by which these drugs increase the amount of functional CFTR protein at the cell surface, which is crucial for proper lung function. By examining the interactions between these drugs and the CFTR protein, researchers hope to uncover new insights that could lead to better treatments for patients with this mutation. The research will utilize advanced techniques to analyze the effects of various CFTR correctors on the protein's stability and activity.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystic fibrosis who carry the ΔF508 mutation.
Not a fit: Patients without the ΔF508 mutation or those with other forms of cystic fibrosis may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective therapies for cystic fibrosis patients with the ΔF508 mutation, potentially improving their lung function and quality of life.
How similar studies have performed: Previous research has shown promise with CFTR correctors in improving outcomes for cystic fibrosis patients, but the specific mechanisms remain underexplored.
Where this research is happening
LOS ANGELES, UNITED STATES
- CEDARS-SINAI MEDICAL CENTER — LOS ANGELES, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: NAREN, ANJAPARAVANDA P — CEDARS-SINAI MEDICAL CENTER
- Study coordinator: NAREN, ANJAPARAVANDA P
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.