title: Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice.
abstract: Human cystic fibrosis transmembrane conductance regulator (CFTR) was expressed in transgenic mice under the control of transcriptional elements derived from the human surfactant protein C (SP-C) gene. The hCFTR mRNA was expressed in lungs and testes: in the lung, we found hCFTR mRNA in bronchiolar and alveolar epithelial cells, and CFTR protein in respiratory epithelial cells. While the level of expression of hCFTR mRNA varied, hCFTR mRNA and protein were detected in pulmonary epithelial cells of several lines. Lung weight, morphology, somatic growth and reproductive capacity were not altered by expression hCFTR in lung and testes of the transgenics. Our findings suggest that hCFTR can be safely transferred to lung epithelial cells for CF therapy.
authors.affiliation: Division of Pulmonary Biology, Children's Hospital, Cincinnati, Ohio 45229-2899.
authors.forename: J A
authors.initials: JA
authors.lastname: Whitsett
authors.forename: C R
authors.initials: CR
authors.lastname: Dey
authors.forename: B R
authors.initials: BR
authors.lastname: Stripp
authors.forename: K A
authors.initials: KA
authors.lastname: Wikenheiser
authors.forename: J C
authors.initials: JC
authors.lastname: Clark
authors.forename: S E
authors.initials: SE
authors.lastname: Wert
authors.forename: R J
authors.initials: RJ
authors.lastname: Gregory
authors.forename: A E
authors.initials: AE
authors.lastname: Smith
authors.forename: J A
authors.initials: JA
authors.lastname: Cohn
authors.forename: J M
authors.initials: JM
authors.lastname: Wilson
mesh_terms: D000818:Animals; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D004848:Epithelium / Q000378:metabolism; D015870:Gene Expression; D015316:Genetic Therapy; D006801:Humans; D007150:Immunohistochemistry; D017403:In Situ Hybridization; D008168:Lung / Q000378:metabolism*; D008297:Male; D008565:Membrane Proteins / Q000096:biosynthesis / Q000235:genetics*; D051379:Mice; D008822:Mice, Transgenic; D011510:Proteolipids / Q000235:genetics; D011663:Pulmonary Surfactants / Q000235:genetics; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism; D014018:Tissue Distribution
chemical_list: C505032:CFTR protein, human; D008565:Membrane Proteins; D011510:Proteolipids; D011663:Pulmonary Surfactants; D012333:RNA, Messenger; D019005:Cystic Fibrosis Transmembrane Conductance Regulator