Article
CFTR Modulator Response in Nasal Organoids Derived from People with Cystic Fibrosis.
Cells - 2 Dec 2025
Lo Cicero Stefania, Castelli Germana, Ceci Aurora, Cerio Anna Maria, Blaconà Giovanna, Virgulti Mariarita, Allushi Sara, Sette Giovanni, Spadaro Francesca, Amato Felice, Melotti Paola, Sorio Claudio, Cimino Giuseppe, Biffoni Mauro, Lucarelli Marco, Eramo Adriana
Abstract excerpt
Despite the progressive extension of CFTR variant eligibility to the triple combination of elexacaftor/tezacaftor/ivacaftor (ETI), most rare CFTR pathogenic variants remain ineligible for CFTR modulators. It is crucial to determine whether unexplored variants are rescuable by clinical modulators and to identify innovative therapeutic strategies for rescuing non-responder variants. The approach known as...
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