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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
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Detection of CFTR function and modulation in primary human nasal cell spheroids
John J Brewington1, Erin T Filbrandt1, F J LaRosa1
1Division of Pulmonary Medicine, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue/MLC 2021, Cincinnati, OH, USA.
Summary
Patient-derived nasal epithelial cell (NEC) spheroids can accurately measure cystic fibrosis transmembrane conductance regulator (CFTR) function and drug response. This facilitates personalized modulator therapy for rare CFTR mutations.
Area of Science:
- Biomedical Engineering
- Cell Biology
- Genetics
Background:
- Cystic Fibrosis (CF) patient-derived models are crucial for expanding CFTR modulator therapies to individuals with rare or undescribed mutations.
- Personalized medicine approaches require accurate quantification of CFTR function and its restoration.
Purpose of the Study:
- To develop a personalized model system for assessing CFTR function and modulator efficacy.
- To utilize non-surgically obtained nasal epithelial cells (NECs) for this model.
Main Methods:
- Nasal epithelial cells (NECs) from healthy volunteers and CF patients were collected via curettage and expanded.
- Cells were cultured in 3D as spheroids, characterized, and stimulated with cAMP-inducing agents to assess CFTR activity.
- Spheroid swelling was quantified as a functional readout of CFTR activity.
Main Results:
- NEC spheroids successfully mimicked pseudostratified respiratory epithelia.
- Spheroid swelling correlated with functional CFTR presence, while shrinkage indicated absence.
- The model accurately quantified CFTR modulator-induced restoration in F508del homozygous CF cells.
Conclusions:
- NEC spheroids represent a promising platform for studying rare CFTR mutations.
- This model enables personalized CFTR modulator testing for diverse patient populations.
- Facilitates evaluation and treatment for CF patients with common, rare, or undescribed mutations.
