Synthesis of Amio Ester Dendrimers as Non-viral Gene Delivery Vectors

Abstract

Gene therapy has been considered as a promising method for the treatment of diseases with genetic defects as well as for the prevention and treatment of acquired diseases. Many researches in gene therapy have focused on the development of reliable delivery systems. It is well agreed that the development of an efficient and safe delivery system to carry the therapeutic genes to a targeted place is the major barrier to the success of current gene therapy. Dendrimers have well-defined, accurately controlled structures and are superior to other non-viral vectors in terms of control over the size and the structure. In addition, dendrimer has many terminal groups which allow further functionalization of the dendrimer and make the dendrimer more versatile. In this study, we developed a novel biodegradable amino ester dendrimer as an efficient non-viral gene delivery vector. Dendrimer was synthesized up to 4th generation by convergent method with a reasonable yield in growing each generation. Primary amino groups were introduced into the terminal groups to condense negatively charged DNA plasmid and protonable tertiary amine was placed in the internal growing units to provide endosomal buffering effect. Also, ester groups in the internal growing units of amino ester dendrimer are expected to show biodegradation. These characteristics make amino ester dendrimer a good candidate as a non-viral gene delivery carrier for gene therapy.