Functional, Cohort-Level Assessment of CFTR Modulator Responses Using Biobanked Nasal Epithelial Cells from Individuals with Cystic Fibrosis.

ABSTRACT: Background/Objectives: Individual responses to CFTR modulators vary widely among people with cystic fibrosis (pwCF), underscoring the need for functional approaches that provide biological context alongside genotype-based therapy selection. Nasal epithelial cultures provide an individual-specific model for theratyping, but most studies rely on freshly isolated cells, restricting repeated testing and long-term sample use. In this study, we tested whether CFTR modulator responses measured in biobanked nasal cells were associated with real-world clinical outcomes. Methods: Cryopreserved nasal epithelial cells from 23 pwCF were differentiated at the air-liquid interface and assessed for CFTR modulator-responsive ion transport using Ussing chambers. In vitro responses were

SUBMITTER: Aalbers BL 

PROVIDER: S-EPMC12842762 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature