Section Reviews: Biologicals & Immunologicals: Recent advances in non-viral gene therapy

Abstract

Gene therapy is beginning to emerge as a safe and potentially effective means to treat human disease, and offers new opportunities to tackle conditions for which no satisfactory treatments have yet been developed. Current approaches rely upon either viral or non-viral vehicles to deliver the therapeutic gene, however, despite encouraging clinical data, it is realistic to expect that further advances are required if any are to be developed, manufactured and used in clinical practice. Non-viral gene therapy is attractive because, in general, it offers the potential to developed a synthetic, generic delivery vehicle, as well as safety and simplicity. However, a host of biological barriers exist, such as cell targeting and entry, intracellular trafficking, and maintenance and controlled expression of the therapeutic gene, which need to be overcome. Recently, non-viral gene therapy research has been an area of considerable innovation, with significant progress being made to address these issues. This review focuses on recent and potential advances to non-viral gene delivery systems and the strategies to ensure therapeutic genes are maintained and expressed in vivo Although further basic research is required, non-viral approaches promise to be an integral, if not major, component of future human gene therapy practice.

Key Words:

• cationic lipids
• direct gene transfer
• gene expression
• genetic immunisation
• gene maintenance
• immuno-tolerance
• liposomes
• non-viral gene therapy
• receptor-mediated gene delivery
• targeted gene delivery
• viral mimetics