Recent Advances in RNA Therapeutics and RNA Delivery Systems Based on Nanoparticles

Abstract Recently, there have been significant advances in the field of RNA‐based therapeutics harnessing exogenous RNAs including small interfering RNA (siRNA), microRNA (miRNA) mimics, and in vitro transcribed (IVT) mRNAs. RNA‐based therapeutics are one of the most attractive classes of drugs for treating a variety of diseases. In principle, RNA‐based therapeutics offer numerous advantages compared to the conventional small‐molecule‐based therapies. However, there are several drawbacks to overcome in the application of RNA‐based therapeutics, including the instability of RNAs and presence of various biological barriers. In the past decades, there have been various attempts to overcome these key challenges. Here, the authors present an overview of RNA‐based therapeutics and recent advances including their delivery systems and clinical applications. In addition, the current challenges of RNA‐based drugs are discussed, followed by perspectives with regard to the development of these promising drug candidates to elicit successful clinical outcomes.