Human Amnion Epithelial Cells Induced to Express Functional Cystic Fibrosis Transmembrane Conductance Regulator
- Monash Univ., Melbourne, VIC (Australia). Monash Inst. for Medical Research. The Ritchie Centre; DOE/OSTI
- Monash Univ., Melbourne, VIC (Australia). Monash Inst. for Medical Research. The Ritchie Centre; Monash Univ., Melbourne, VIC (Australia). Dept. of Obstetrics and Gynaecology
- Monash Univ., Melbourne, VIC (Australia). School of Chemistry. Centre for Biospectroscopy
- Monash Univ., Melbourne, VIC (Australia). Monash Centre for Synchrotron Science
- Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States). Physical Biosciences Division
- Australian Synchrotron, Melbourne, VIC (Australia). X-ray Fluorescence Microscopy
- Monash Univ., Melbourne, VIC (Australia). Monash Inst. for Medical Research. The Ritchie Centre
- Wake Forest Univ., Winston-Salem, NC (United States). School of Medicine. Wake Forest Inst. for Regenerative Medicine
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 (I2/ Cl2) 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.
- Research Organization:
- Lawrence Berkeley National Laboratory (LBNL), Berkeley, CA (United States)
- Sponsoring Organization:
- USDOE Office of Science (SC), Biological and Environmental Research (BER). Biological Systems Science Division
- Grant/Contract Number:
- AC02-05CH11231
- OSTI ID:
- 1627554
- Journal Information:
- PLoS ONE, Journal Name: PLoS ONE Journal Issue: 9 Vol. 7; ISSN 1932-6203
- Publisher:
- Public Library of ScienceCopyright Statement
- Country of Publication:
- United States
- Language:
- English
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