Preorganized, Macromolecular, Gene-Delivery Systems

Viruses represent a paradigmatic example of multicomponent, self-organized supramolecular systems specialized in the delivery and replication of their genetic material. Mimicking their functioning by artificial synthetic molecules represents a fantastic challenge that will lead to the future development of gene therapy. This is only possible if general approaches towards the construction of nanoscale vehicles for DNA are developed and the key rules governing their capacity to compact genetic material and its active transport/delivery through cell membranes are understood. In this area of research, synthetic organic chemistry plays an important role by providing tools to create tailor-made molecules of increasing complexity. Preorganization of functional elements onto macromolecular platforms has the potential to allow control of the self-assembling behavior of discrete architectures to produce nanometric objects that can be programmed to complex, compact, deliver, and release plasmid DNA in a target cell.