Publications by authors named "Erin T Filbrandt"
Recent advances in the management of cystic fibrosis (CF) target underlying defects in the CF transmembrane conductance regulator (CFTR) protein, but efficacy analyses remain limited to specific genotype-based subgroups. Patient-derived model systems may therefore aid in expanding access to these drugs. Brushed human nasal epithelial cells (HNEs) are an attractive tissue source, but it remains unclear how faithfully they recapitulate human bronchial epithelial cell (HBE) CFTR activity.
View Article and Find Full Text PDFArticle Synopsis
- The introduction of CFTR modulator drugs has improved Cystic Fibrosis treatment, but current genotype-directed therapies exclude certain mutations and make individualized optimization challenging.
- Human nasal epithelial cells (HNEs) provide a promising minimally invasive source for creating a three-dimensional spheroid model that can assess CFTR function and modulation.
- This model allows for personalized evaluation of CFTR activity, with ongoing studies aimed at linking lab results to actual patient responses to treatment.
Background: Expansion of CFTR modulators to patients with rare/undescribed mutations will be facilitated by patient-derived models quantifying CFTR function and restoration. We aimed to generate a personalized model system of CFTR function and modulation using non-surgically obtained nasal epithelial cells (NECs).
Methods: NECs obtained by curettage from healthy volunteers and CF patients were expanded and grown in 3-dimensional culture as spheroids, characterized, and stimulated with cAMP-inducing agents to activate CFTR.