Synthetic vectors and gene therapy

Synthetic vectors for gene delivery have gained widespread interest and use since the introduction of the first cationic lipids about ten years ago, More and more studies are accumulating, in the effect to exploit the advantages and to overcome the Limitations presented by this approach over viral methods. Italian as well as international reports are reviewed in this chapter. The structure of transfection-competent molecules, mainly cationic lipids or polycations, and the methods used to assess transfection efficiency are presented, and aspects of DNA plasmid design and assembly are reported. Examples of non-viral in vitro gene delivery, in particular to the lung and to the kidney, are described, Finally, methods to study the physico-chemical properties of the DNA-containing particles and the molecular mechanisms involved in the intracellular trafficking required for successful gene expression are discussed.