Non-viral Gene Therapy

Abstract

Gene therapy has been considered to be a powerful approach for the prevention and/or treatment of a variety of diseases from genetic disorders, infections, to cancer. The success of gene therapy in the clinic is largely limited currently, mainly due to the lack of safe and efficient delivery vectors. Despite the high transfection efficiency, viral vectors encounter the vital toxicity issues and production problems. Increasing endeavors have been therefore directed towards the development of non-viral systems with the advantages of low immunogenicity and toxicity, ease in manufacturing and mass production, low cost, excellent stability, reduced vector size limitations, high flexibility regarding the size of transgenes to be delivered, and diverse chemical designs for constructing vectors with multiple functions. In this chapter, we summarized most of the synthetic non-viral systems currently employed for gene therapy, including lipid and polymer-based vectors, nanomaterials such as magnetic nanoparticles, quantum dots, gold/silica nanostructures, carbon nanotubes, calcium phosphate nanoparticles, and layered double hydroxides/clays, as well as multifunctional nanosystems among them. Particular attention has been paid on synthetic polymers and the related nanomedicines. Selected clinical trials of gene therapy using non-viral vectors as well as the future development in this rapidly growing field were briefly discussed.