Nanostructures for oral delivery of therapeutic nucleic acids

The promise of using nucleic acid as a drug has opened new therapeutics strategy for a large variety of diseases. Several types of therapeutic nucleic acid exist, ranging from short synthetic RNA- or DNA-based oligonucleotides to plasmid-based DNA-expressing constructs. According to their functional activity, they can either induce the expression of a protein (gene therapy strategy) or trigger the silencing of a gene (RNA interference or antisense strategy). Current nucleic acid–based treatment strategies are largely injection-based, but effective oral delivery platforms for nucleic acids may result in improved patient comfort and compliance. In this chapter, we propose to discuss the barriers to oral delivery of nucleic acids as well as review the various technologies already tested in preclinical study and the experimental models available to assay oral delivery systems.