CATIONIC LIPOSOME-MEDIATED GENE-TRANSFER

Direct gene transfer for the treatment of human diseases requires a ve ctor which can be administered efficiently, safely and repeatedly. Cat ionic liposomes represent one of the few examples that can meet these requirements. Currently, more than a dozen cationic liposome formulati ons have been reported. These liposomes bind and condense DNA spontane ously to form complexes with high affinity to cell membranes. Endocyto sis of the complexes followed by disruption of the endosomal membrane appears to be the major mechanisms of gene delivery. The effectiveness and safety of this DNA delivery method has been established in many s tudies. Based on these results, two human gene therapy clinical trials using cationic liposomes have been conducted and more trials will be started in the near future. The simplicity, efficiency and safety feat ures have rendered the cationic liposome an attractive vehicle for hum an gene therapy.