Human Amnion Epithelial Cells Induced to Express Functional Cystic Fibrosis Transmembrane Conductance Regulator
Year: 2012
Type: article
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.
Source: PLoS ONE
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,382
Funders: U.S. Department of Energy, Cystic Fibrosis in Australia, Australian Synchrotron, Australian Cystic Fibrosis Research Trust, State Government of Victoria, National Institutes of Health, National Health and Medical Research Council, National Institute of General Medical Sciences, National Center for Research Resources, Biological and Environmental Research, Lawrence Berkeley National Laboratory
Awards: G1079818582, G1143651532, G1245477126, G1286236842, G1337544421, Resource for X-Ray Tomography of Whole Cells, G1581387393, G1816284028, G223002938, G2539837713, G2857863752, G3083819904, G3432487278, G3557443024, G365575497, G382641700, G42916606, G4959774877, Preventing preterm lung disease., G654302390, G7354628648, G7757261926, G7787287669, G8659670254, G969889393
OpenAlex ID: W2083978086