Design of new gene delivery carriers
Non-viral genetic therapies are not only employed in the cure of genetic disorders, but additionally in vaccination or allergy treatment. The expansion of the operation range arose with the discovery of RNA interference (RNAi), a mechanism for a sequence-specific inhibition of target genes, which can be therapeutically used for the treatment of numerous diseases.
However, there are physicochemical and biological barriers that hinder the delivery of free oligo- or polynucleotides. Therefore, their use relies on suitable systems to achieve an efficient, safe and clinically appropriate delivery to overcome biological barriers.
New formulations from current approaches in polymer chemistry and nanotechnology are developed. In these formulations, the drug is either bound coordinatively (polyrotaxanes complexing acid polymers) or encapsulated in nanoparticles (core-shell carrier particles based on biodegradable polymers like chitosan-coated PLGA particles).
The aim is the development of carrier systems that can be adapted to various application routes by adjustable physical and pharmacokinetic properties. Such transport systems are optimized for the intake via the lung or via the intestinal tract.
- Drug Delivery - Prof. Dr. Claus-Michael Lehr