CHAPTER 13: Cationic Dendritic Systems as Non-viral Vehicles for Gene Delivery Applications

The possibility to use nucleic acids as therapeutics requires adequate carriers that can guide them to the cell. In this way, the physical and chemical properties of dendritic macromolecules make them very attractive for this purpose. As nucleic acids are negatively charged, cationic dendrimers, and related systems, are being employed as gene delivery vectors due to their ability to form complex dendrimer/nucleic acid (dendriplexes) through electrostatic interactions. In this chapter we survey the main type of dendritic molecules and describe general synthetic procedures for cationic dendrimers. The diversity of structures, their macromolecular nature, the multivalency, the cationic charge and other features of cationic dendrimers that involve their characterization can be confronted using a wide variety of techniques. The last section of the chapter deals with applications of dendrimers in gene therapy, discussing their main advantages and presenting both in vitro and in vivo assays.