30May 2025
Lumacaftor Corrects CFTR but Inhibits Channel Activity in Cells

Lumacaftor Corrects CFTR but Inhibits Channel Activity in Cells

The study reveals that while lumacaftor improves F508del CFTR processing, it paradoxically inhibits its channel activity. Real-time assays and patch-clamp data show reduced open probability and forskolin-induced currents in CFBE-DF and HEK-DF cells. Unlike tezacaftor or elexacaftor, lumacaftor disrupts functional rescue at 37°C, possibly explaining Orkambi’s modest clinical performance.

  • #pulmonology

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