Nanomaterials for Therapeutic Nucleic Acid Delivery

Nucleic acids and their chemically modified derivatives have gradually become an important class of therapeutic agents. Several nucleic acid-based drugs have been applied to treat various diseases at the genetic level by regulating gene expression and protein translation. Despite these breakthroughs, delivery of nucleic acids to desired tissues and cell populations remains a challenge for clinical translation. To address the delivery issues, researchers have investigated many types of nanomaterials to encapsulate nucleic acids and overcome physiological barriers. Commonly used nanomaterials include polymers, lipids, cellpenetrating peptides (CPPs), inorganic nanomaterials, nucleic acid-based nanoparticles, and viruslike particles (VLPs), several of which have been approved for clinical use. This chapter presents an overview of therapeutic nucleic acids, highlights chemical modification strategies, and introduces representative nanomaterials which may facilitate clinical translation of therapeutic nucleic acids.