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
Drugs with this mechanism (1)
- Ivacaftor — The first CFTR potentiator; opens gating-mutation channels already at the surface.
Self-check
- Which CFTR gating mutation exemplifies benefit from potentiation?
G551D. - For F508del, what is paired with a CFTR potentiator?
Correctors.
All mechanisms · Open CFTR potentiator in the interactive console ↗