Recombinant CTFR detection in CF tracheal epithelial cells following in vitro liposomeme-mediated gene transfer.

Abstract

The efficacy of CFTR gene transfer mediated by cationic liposomes Dc-Chol/DOPE into cystic fibrosis (CF) tracheal epithelial cells carrying defective processing mutations (S549N/N1303K), was assessed by studying mRNA and protein expression of the recombinant product. Appreciable levels of mRNA transcripts were detected 48 h after transfection, while complete translocation of the recombinant CFTR to the apical membrane of epithelial cells was observed after 72 h following transfection. Our results suggest that in vitro restoration of a normal CFTR processing and migration to the cell plasmalemma requires 72 h at least as demonstrated by immunocyto-fluorescence using the monoclonal antibody MATG 1016. These findings are relevant onto gene transfer phase I clinical studies.

• Authors: A Colosimo, S Scarpino, F Sangiuolo, S D Sario, G Mossa, G Novelli, B Dallapiccola
• Journal: Biochemistry and molecular biology international (Biochem Mol Biol Int) Vol. 42 Issue 4 Pg. 723-9 (Jul 1997) ISSN: 1039-9712 [Print] England
• PMID: 19856289 (Publication Type: Journal Article)