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.

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nasal turbinate apical epithelial hnecs gene therapy vectors chloridebicarbonate channel human nasal epithelial cultures cf transmembrane conductance regulator cftr gene limiting helperdependent adenoviral hdad vector

Jing Wu,Huibi Cao,Hong Ouyang,Wan Ip,Kai Du,Wenming Duan,Julie Avolio,Cathleen Duan,Herman Yeger,Christine E Bear,Tanja Gonska,Jim Hu,Theo J Moraes,.Testing gene therapy vectors in human primary nasal epithelial cultures. 2 (C),.

Jing Wu,Huibi Cao,Hong Ouyang,Wan Ip,Kai Du,Wenming Duan,Julie Avolio,Cathleen Duan,Herman Yeger,Christine E Bear,Tanja Gonska,Jim Hu,Theo J Moraes,"Testing gene therapy vectors in human primary nasal epithelial cultures" 2

Jing Wu,Huibi Cao,Hong Ouyang,Wan Ip,Kai Du,Wenming Duan,Julie Avolio,Cathleen Duan,Herman Yeger,Christine E Bear,Tanja Gonska,Jim Hu,Theo J Moraes,"Testing gene therapy vectors in human primary nasal epithelial cultures"