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Target intelligence / Profile preview
Cystic Fibrosis Transmembrane Conductance Regulator Chloride Channel (CFTR)
- Target
- CFTR
- Molecular classification
- ABC transporter, Ion channel, Transporter
01
Overview
The cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and chloride channel that regulates the movement of chloride and bicarbonate ions across epithelial cell membranes. Mutations in the *CFTR* gene cause cystic fibrosis, a genetic disorder characterized by thickened secretions in the lungs, pancreas, and other organs.
02
Mechanism of action
CFTR potentiators enhance the channel-opening probability (gating) of mutant CFTR proteins at the cell surface. CFTR correctors promote the proper folding and trafficking of misfolded CFTR proteins to the cell surface.
03
Biological functions
Chloride ion transportBicarbonate ion transportRegulation of epithelial fluid balanceRegulation of mucus consistency
04
Disease associations
Cystic fibrosisSecretory diarrheaPolycystic kidney disease
05
Safety considerations
Drug interactionsAdverse effects of CFTR modulators (e.g., elevated liver enzymes)Limited efficacy in certain CFTR mutationsHigh cost of CFTR modulator therapies
06
Interacting drugs
CFTR potentiators (e.g., Ivacaftor)
1 more in the full profile.
07
Biomarkers
Sweat chloride testCFTR genotypingNasal potential difference (NPD)
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