CFTR potentiator
CFTR channel potentiator
- Holds surface CFTR (cystic fibrosis transmembrane conductance regulator) channels open → ↑ chloride transport
- Transforms disease in gating mutations (G551D)
- Paired with correctors for F508del
- Effect depends entirely on the specific CFTR defect present
- Increases chloride transport through defective CFTR
- Effect depends on which CFTR defect is present
Drugs with this mechanism (1)
- Ivacaftor — The first CFTR potentiator; opens gating-mutation channels already at the surface.
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