Towards Defined DNA and RNA Delivery Vehicles Using Nucleic Acid Nanotechnology

Both DNA and RNA nanostructures show exceptional programmability, modularity, and self-assembly ability. Using DNA or RNA molecules it is possible to assemble monodisperse particles that are homogeneous in size and shape and with identical positioning of surface modifications. For therapeutic applications such nanoparticles are of particular interest as they can be tailored to target cells and reduce unwanted side effects due to particle heterogeneity. Recently, DNA and RNA nanostructures have demonstrated this potential by delivery of drugs to specific cells in vitro and in vivo. This has launched an increasing interest to engineer-defined DNA and RNA vehicles for drug delivery. However, before this can be realized, key challenges must be overcome including structure integrity, efficient cell targeting, and drug release. The tunable nature of DNA and RNA assemblies allows for thorough investigations into various structural and functional features, which can address these challenges. To facilitate the synthesis process novel methods enable the construction of sophisticated structures and attachment of relevant functionalities. In this chapter we will discuss the state-of-the-art molecular designs and approaches to harness and use DNA and RNA nanostructures in drug delivery.