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Kollen, WJW ; Midoux, P ; Erbacher, P ; Yip, A ; Roche, AC ; Monsigny, M ; Glick, MC ; Scanlin, TF

Gluconoylated and glycosylated polylysines as vectors for gene transfer into cystic fibrosis airway epithelial cells

Human Gene Therapy 7 (13) 1577-1586

par Administrateur - publié le

Abstract :

To provide an alternative to viral vectors for the transfer of genes into airway epithelial cells in cystic fibrosis (CF), a novel set of substituted polylysines were employed, Polylysine was partially neutralized by blocking a number of positively charged residues with gluconoyl groups, In addition, polylysine was substituted with sugar residues on a specified number of amino groups, Using the gluconoylated polylysine as vector, the pCMVLuc plasmid gave high expression of the reporter gene luciferase in immortalized CF/T43 cells. The luciferase activity was 75-fold greater in the presence of 100 mu M chloroquine. Luciferase gene expression persisted at high levels for up to at least 120 hr following transfection.