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Effect of vanzacaftor/tezacaftor/ivacaftor on cystic fibrosis nasal epithelial cells and intestinal organoids compared to elexacaftor/tezacaftor/ivacaftor - PubMed

6 hours ago
  • #CFTR modulator therapy
  • #cystic fibrosis
  • #nasal epithelial cells
  • The study compares the effects of vanzacaftor/tezacaftor/ivacaftor (VAN/TEZ/IVA) and elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cystic fibrosis (CF) nasal epithelial cells and intestinal organoids.
  • VAN/TEZ/IVA restored CFTR function to 67% of wild-type levels in nasal epithelial cells, compared to 45% with ELX/TEZ/IVA.
  • Both triple combinations showed comparable effects on intestinal organoids using the forskolin-induced swelling (FIS) assay.
  • The study highlights the superior discriminatory power of airway epithelial electrophysiology over intestinal organoids for comparing CFTR modulator therapies.
  • VAN/TEZ/IVA demonstrated greater CFTR functional rescue in F508del/F508del nasal epithelial cells than ELX/TEZ/IVA.