An expanding protocol to validate nasospheroids as a functional assay to study airway CFTR-fluid transport

We aimed to study CF patient derived cells to determine CFTR functionality and drug response. Intestinal organoids is a validated and accepted method to measure the preclinical individual efficacy of CFTR modulators. However, this technique is time consuming and requires hands-on experience. Therefore, we investigated alternative methods and cells to study CFTR function. The aim of our study was to validate the previously reported methodology of nasospheroids (Guimbellot Jet al 2017) and to apply this technique to study CFTR functionality and predict responses of cystic fibrosis patients to different drugs.

Nasospheroids from 7 controls and 7 CF patients presenting different genotypes were established in culture for ≤10 days. Cells were studied by live-cell microscopy. To validate our results, a statistical equation was developed to obtain personalized mixed linear models (R v3.1.2 software)