title: Testing gene therapy vectors in human primary nasal epithelial cultures.
abstract: Cystic fibrosis (CF) results from mutations in the CF transmembrane conductance regulator (CFTR) gene, which codes for a chloride/bicarbonate channel in the apical epithelial membranes. CFTR dysfunction results in a multisystem disease including the development of life limiting lung disease. The possibility of a cure for CF by replacing defective CFTR has led to different approaches for CF gene therapy; all of which ultimately have to be tested in preclinical model systems. Primary human nasal epithelial cultures (HNECs) derived from nasal turbinate brushing were used to test the efficiency of a helper-dependent adenoviral (HD-Ad) vector expressing CFTR. HD-Ad-CFTR transduction resulted in functional expression of CFTR at the apical membrane in nasal epithelial cells obtained from CF patients. These results suggest that HNECs can be used for preclinical testing of gene therapy vectors in CF.
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Huibi
authors.initials: H
authors.lastname: Cao
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Hong
authors.initials: H
authors.lastname: Ouyang
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Wan
authors.initials: W
authors.lastname: Ip
authors.affiliation: Programme in Molecular Structure & Function, Research Institute, Hospital for Sick Children , Toronto, Ontario, Canada.
authors.forename: Kai
authors.initials: K
authors.lastname: Du
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Wenming
authors.initials: W
authors.lastname: Duan
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Julie
authors.initials: J
authors.lastname: Avolio
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Jing
authors.initials: J
authors.lastname: Wu
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Cathleen
authors.initials: C
authors.lastname: Duan
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children , Toronto, Onatrio, Canada.
authors.forename: Herman
authors.initials: H
authors.lastname: Yeger
authors.affiliation: Programme in Molecular Structure & Function, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada; Department of Biochemistry, University of Toronto, Toronto, Ontario, Canada; Department of Physiology, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Christine E
authors.initials: CE
authors.lastname: Bear
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children, Toronto, Onatrio, Canada; Department of Paediatrics, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Tanja
authors.initials: T
authors.lastname: Gonska
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children, Toronto, Onatrio, Canada; Programme in Molecular Structure & Function, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Jim
authors.initials: J
authors.lastname: Hu
authors.affiliation: Programme in Physiology & Experimental Medicine, Research Institute, Hospital for Sick Children, Toronto, Onatrio, Canada; Department of Paediatrics, University of Toronto, Toronto, Ontario, Canada; Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Theo J
authors.initials: TJ
authors.lastname: Moraes