Pharmaceutical perspectives of nonviral gene therapy

The use of nonviral plasmid-based gene medicines represents an attractive in vivo gene transfer strategy that is simple and lacks many risks that are inherent to viral systems. Commercialization of gene medicines requires a thorough analysis of business opportunities, unmet clinical needs, competitive products under development, and issues related to intellectual property. Synthetic gene delivery systems are designed to control the location of a gene within the body by affecting distribution and access of a gene expression system to the target cell, and/or recognition by a cell surface receptor and uptake followed by intracellular and nuclear translocation. Plasmid-based gene expression systems are designed to control the level, fidelity, and duration of in vivo production of a therapeutic gene product. This review will provide insights into the potentials of plasmid-based gene therapy and critical evaluation of gene delivery sciences and clinical applications of gene medicines.