Current Progress and Future Prospects in Nucleic Acid Based Therapeutics

Abstract

Functional nucleic acids have been gaining escalated attention and extensive research has been performed for the purpose of developing this new class of therapeutics. The challenges in development of nucleic acid based therapeutics include their extremely unstable nature in biological fluid, poor pharmacokinetic (PK) properties, and potential activation of innate immunity. Gaining a better understanding of target biology, PK properties, and functional pathways of therapeutic agents will facilitate approaches to improve their properties, and thus enhance clinical development of nucleic acid based therapeutics. In this chapter we discuss the current advances and potential challenges in the development of synthetic and biochemically produced nucleic acid therapeutics along with mechanisms of action for each technology.