Publication Type	Academic Article
Authors	Rosenfeld M, Yoshimura K, Trapnell B, Yoneyama K, Rosenthal E, Dalemans W, Fukayama M, Bargon J, Stier L, Stratford-Perricaudet L
Journal	Cell
Volume	68
Issue	1
Pagination	143-55
Date Published	01/10/1992
ISSN	0092-8674
Keywords	Cystic Fibrosis, Lung, Membrane Proteins, Transfection
Abstract	Direct transfer of the normal cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene to airway epithelium was evaluated using a replication-deficient recombinant adenovirus (Ad) vector containing normal human CFTR cDNA (Ad-CFTR). In vitro Ad-CFTR-infected CFPAC-1 CF epithelial cells expressed human CFTR mRNA and protein and demonstrated correction of defective cAMP-mediated Cl- permeability. Two days after in vivo intratracheal introduction of Ad-CFTR in cotton rats, in situ analysis demonstrated human CFTR gene expression in lung epithelium. PCR amplification of reverse transcribed lung RNA demonstrated human CFTR transcripts derived from Ad-CFTR, and Northern analysis of lung RNA revealed human CFTR transcripts for up to 6 weeks. Human CFTR protein was detected in epithelial cells using anti-human CFTR antibody 11-14 days after infection. While the safety and effectiveness remain to be demonstrated, these observations suggest the feasibility of in vivo CFTR gene transfer as therapy for the pulmonary manifestations of CF.
DOI	10.1016/0092-8674(92)90213-v
PubMed ID	1370653