Nanoarchitectures to Deliver Nucleic Acid Drugs to Disease Sites

Abstract Therapeutic nucleic acid drugs (antisense DNA, siRNA, DNAzyme, and others) have been widely employed to regulate the expression of a disease‐causing gene. For the correction of a gene, the CRISPR/Cas9 system is available. Advantageously, they can be straightforwardly designed in terms of the Watson‐Crick rule. In order to deliver these nucleic acid drugs to a target place in our body (organs and cells) at desired timing, various nanoarchitectures have been elegantly designed. Their primary roles are protection of drugs from degradation, increase of cell‐membrane permeability, and spatiotemporal control of delivery. Encapsulated nucleic acid drugs are released by decomposing the nanostructures with either external stimuli or intracellular signals. Furthermore, therapeutic efficiency is enhanced by simultaneously delivering multiple types of nucleic acid drugs for their cooperation. Composites of nanoarchitectures and therapeutic nucleic acid drugs are highly promising for future applications.