Emergence of Small Nucleic Acids as Drugs in Precision Medicine

Small nucleic acid drugs including antisense oligonucleotides, small interfering RNAs, microRNAs, and aptamers, among others, have revolutionized the pharmaceutical industry in the 21st century, offering novel therapeutic strategies for a myriad of diseases through precise gene expression regulation or protein synthesis modulation. As of December 2024, 19 small nucleic acid drugs have been approved globally, with applications primarily in treating rare and hereditary conditions, metabolic diseases, and ophthalmological disorders, demonstrating great clinical potential. This review provides an overview of the classification, mechanism of action, technical challenges, and addressing strategies for small nucleic acid drugs, with a focus on the indications for market-available drugs, aiming to equip healthcare professionals with a thorough understanding and practical guidance on their utilization.