Gene Therapy with Plasmid DNA

Abstract Recent advances in the study of human genomics have revealed the relationship between somatic aberrations and cancer. Therapeutic genetic manipulation for cancer therapy can be achieved through treatment regimens that include gene therapy. Messenger RNA and plasmid DNA are widely applied in gene therapy. Messenger RNA therapy has received much attention in the past 10 years. In this article, we first compare messenger RNA therapeutics with plasmid DNA therapeutics and go on to list the limitations of messenger RNA therapy and advantages of plasmid DNA therapeutics. To address the unresolved challenges for plasmid DNA for further translational application, this article describes the present clinical usage of plasmid DNA, reviews the current physical and chemical delivery system proposed for nonviral plasmid DNA therapy, discusses targeted delivery strategies and specific formulation designs with promising outcomes in preclinical studies, and provides future perspectives for plasmid DNA therapy development.