Rationally Designed Synthetic Vectors for Gene Delivery

Abstract

Vector development is one of the most important challenges facing the successful use of genes for treatment of diseases. Although chemically produced vectors offer distinct advantages over biological systems such as viruses, there are still some hurdles that have to be overcome before synthetic gene delivery vectors can be successfully implemented. This brief review discusses the biological barriers that limit current delivery strategies and reviews currently employed strategies for plasmid delivery. Nanoparticle-based gene delivery is reviewed along with methods for their characteriza- tion, physiochemical properties and toxicity. Finally a prospectus is provided for future development of an ideal synthetic gene delivery vector. GENE THERAPY Gene therapy involves the use of exogenous DNA as the therapeutic agent. Originally it was targeted towards treat- ment of inheritable single-gene disorders caused due to an absent or defective gene. However, applications of gene therapy have expanded to include treatment of acquired and infectious diseases and exogenously administered genes now are used in a wide variety of applications including immu- nomodulation, genetic vaccination and genetic pharmacol- ogy (1-6).