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Updated: May 18, 2026
Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
Human amnion epithelial cells induced to express functional cystic fibrosis transmembrane conductance regulator
Sean V Murphy1, Rebecca Lim, Philip Heraud
1The Ritchie Centre, Monash Institute for Medical Research, Monash University, Melbourne, Australia.
Insights
Human amnion epithelial cells express functional cystic fibrosis transmembrane conductance regulator (CFTR) protein and ion channels. These cells show promise for developing a cellular therapy for cystic fibrosis.
Area of Science:
- Cell Biology
- Genetics
- Respiratory Medicine
Background:
- Cystic fibrosis is a genetic disorder impacting lung health due to CFTR gene mutations.
- It causes thick mucus, leading to infections, lung damage, and reduced lifespan.
- No cure currently exists for cystic fibrosis.
Purpose of the Study:
- To investigate if human amnion epithelial cells (hAECs) can express functional CFTR.
- To evaluate hAECs as a potential source for cystic fibrosis cellular therapy.
Main Methods:
- Culturing hAECs in Small Airway Growth Medium (SAGM).
- Assessing CFTR gene and protein expression.
- Analyzing CFTR protein distribution using immunofluorescence.
- Measuring iodide/chloride ion channel activity and inhibition by CFTR-172.
Main Results:
- hAECs formed 3D structures and expressed CFTR gene and protein in SAGM.
- Polarized CFTR distribution was observed on hAEC membranes.
- hAECs exhibited functional iodide/chloride ion channels inhibited by CFTR-172.
Conclusions:
- hAECs express functional CFTR, similar to airway cells.
- hAECs represent a potential cell source for cystic fibrosis therapy development.
Abstract:
Cystic fibrosis, an autosomal recessive disorder caused by a mutation in a gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), remains a leading cause of childhood respiratory morbidity and mortality. The respiratory consequences of cystic fibrosis include the generation of thick, tenacious mucus that impairs lung clearance, predisposing the individual to repeated and persistent infections, progressive lung damage and shortened lifespan. Currently there is no cure for cystic fibrosis. With this in mind, we investigated the ability of human amnion epithelial cells (hAECs) to express functional CFTR. We found that hAECs formed 3-dimensional structures and expressed the CFTR gene and protein after culture in Small Airway Growth Medium (SAGM). We also observed a polarized CFTR distribution on the membrane of hAECs cultured in SAGM, similar to that observed in polarized airway cells in vivo. Further, hAECs induced to express CFTR possessed functional iodide/chloride (I(-/)Cl(-)) ion channels that were inhibited by the CFTR-inhibitor CFTR-172, indicating the presence of functional CFTR ion channels. These data suggest that hAECs may be a promising source for the development of a cellular therapy for cystic fibrosis.
