Therapeutic Option of Plasmid-DNA Based Gene Transfer

Yoshiaki      Taniyama; Junya      Azuma; Yasuo      Kunugiza; Kazuma      Iekushi; Hiromi      Rakugi; Ryuichi      Morishita

doi:10.2174/156802612803531342

Abstract

Gene therapy offers a novel approach for the prevention and treatment of a variety of diseases, but it is not yet a common method in clinical cases because of various problems. Viral vectors show high efficiency of gene transfer, but they have some problems with toxicity and immunity. On the other hand, plasmid deoxyribonucleic acid (DNA)-based gene transfer is very safe, but its efficiency is relatively low. Especially, plasmid DNA gene therapy is used for cardiovascular disease because plasmid DNA transfer is possible for cardiac or skeletal muscle. Clinical angiogenic gene therapy using plasmid DNA gene transfer has been attempted in patients with peripheral artery disease, but a phase III clinical trial did not show sufficient efficiency. In this situation, more efficient plasmid DNA gene transfer is needed all over the world. This review focuses on plasmid DNA gene transfer and its enhancement, including ultrasound with microbubbles, electroporation, hydrodynamic method, gene gun, jet injection, cationic lipids and cationic polymers.

Keywords: Plasmid DNA, peripheral artery disease (PAD), ultrasound, electroporation, hydrodynamic method, ultrasound, microbubble, cavitation, cavitation, Gene Transfer