CFTR function and clinical response to modulators parallel nasal epithelial organoid swelling.

ABSTRACT: In vitro biomarkers to assess cystic fibrosis transmembrane conductance regulator activity are desirable for precision modulator selection and as a tool for clinical trials. Here, we describe an organoid swelling assay derived from human nasal epithelia using commercially available reagents and equipment and an automated imaging process. Cells were collected in nasal brush biopsies, expanded in vitro, and cultured as spherical organoids or as monolayers. Organoids were used in a functional swelling assay with automated measurements and analysis, whereas monolayers were used for short-circuit current measurements to assess ion channel activity. Clinical data were collected from patients on modulators. Relationships between swelling data and short-circuit current, as well as between swelling

SUBMITTER: Anderson JD 

PROVIDER: S-EPMC8321858 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature