ABSTRACT
Ribonucleic acid interference (RNAi) is a sequence-targeted post transcriptional gene-silencing mechanism. This chapter provides a summary of the RNAi mechanism, the challenges and limits of RNAi as therapeutic agents in the in vivo environment and discusses examples of self-assembled nanotechnology platforms for delivery of RNAi reagents. Self-assembly of RNA nanoparticles depends on the spontaneous interaction of individual RNA molecules that base pair in a predefined manner to form 2D or 3D RNA nanostructures. The clinical trials relied on the improved small interfering RNAs (siRNAs) delivery formulations such as polymer nanoparticles and liposomes. Various RNA-based therapeutics such as siRNAs, aptamers, ribozymes and antisense have been successfully linked to an RNA nanoparticle in order to achieve targeted delivery, enhance potency and minimize unwanted side effects. The self-assembled nano-junction is resistant to denaturation in 8M urea, stable in serum and able to carry a wide array of functional moieties such as siRNA, aptamers and receptor ligands like folate.
