L1077P CFTR Pathogenic Variant Function Rescue by Elexacaftor–Tezacaftor–Ivacaftor in Cystic Fibrosis Patient-Derived Air–Liquid Interface (ALI) Cultures and Organoids: In Vitro Guided Personalized Therapy of Non-F508del Patients

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Using the innovative approach of using nasal conditionally reprogrammed cell-based models that mimic patient disease in vitro, investigators theratyped orphan cystic fibrosis transmembrane conductance regulator mutation L1077P.
[Respiratory Research]
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