Gene therapy for central nervous system injury: the use of cationic liposomes: an invited review.

This paper briefly reviews general principles of gene therapy with emphasis on the therapeutic potential of cationic liposome-mediated neurotrophin gene transfer to treat central nervous system (CNS) injury. Current developments in studies of gene therapy for CNS injury are both impressive and promising. Ex vivo gene transfer into the CNS is relatively mature in animal studies following more than a decade of experimental studies. In vivo gene transfer into the CNS has gained more attention recently. Although progress has been made using viral vectors, rapid advances in transfection technologies employing cationic liposomes, together with the relatively low toxicity of these nonviral vector systems, suggest that liposomes may have significant potential for clinical applications. Although many investigators have recognized that gene therapy may be useful for treatment of certain genetic defect diseases or cancer, gene therapy for CNS injury is relatively novel. In contrast to genetic defect disorders, temporary induction of transgenes may have therapeutic applications for CNS injuries such as stroke and trauma. Employing gene transfer techniques to achieve therapeutically useful levels of expression of neurotrophins in the CNS could provide a new strategy for treatment of the traumatically injured CNS.