CATIONIC LIPID-BASED GENE DELIVERY SYSTEMS - PHARMACEUTICAL PERSPECTIVES

Gene delivery systems are designed to control the location of administ ered therapeutic genes within a patient's body. Successful in vivo gen e transfer may require (i) the condensation of plasmid and its protect ion from nuclease degradation, (ii) cellular interaction and internali zation of condensed plasmid, (iii) escape of plasmid from endosomes (i f endocytosis is involved), and (iv) plasmid entry into cell nuclei. E xpression plasmids encoding a therapeutic protein can be, for instance , complexed with cationic liposomes or micelles in order to achieve ef fective in vivo gene transfer. A thorough knowledge of pharmaceutics a nd drug delivery, bio-engineering, as well as cell and molecular biolo gy is required to design optimal systems for gene therapy. This mini-r eview provides a critical discussion on cationic lipid-based gene deli very systems and their possible uses as pharmaceuticals.