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 immunocytofluorescence using the monoclonal antibody MATG 1016. These findings are relevant onto gene transfer phase I clinical studies.
Original language | English |
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Pages (from-to) | 723-729 |
Number of pages | 7 |
Journal | Biochemistry and Molecular Biology International |
Volume | 42 |
Issue number | 4 |
Publication status | Published - Jul 1997 |
Keywords
- Cationic liposomes
- Cystic fibrosis
- Gene therapy
- Immunocytochemistry
- Tracheal epithelial cells
ASJC Scopus subject areas
- Biochemistry
- Genetics
- Molecular Biology