CFTR corrector
- Rescues folding and trafficking, allowing mutant CFTR to reach the cell surface
- Distinct from a POTENTIATOR, which holds an already-surfaced channel open
- Corrector alone does little; the pairing is the therapy
Drugs with this mechanism (2)
- Lumacaftor — CFTR corrector paired with ivacaftor; a strong 3A4 inducer that undercuts its own partner.
- Tezacaftor — CFTR corrector used in cystic-fibrosis combinations; improves mutant protein trafficking rather than directly opening the channel.
Self-check
- What cellular defects do CFTR correctors rescue?
Folding and trafficking to the cell surface. - What does a CFTR potentiator do once a channel reaches the surface?
Holds it open.
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