Fig. 7

The challenges and future directions of small nucleic acid drugs. Various challenges in the development of small nucleic acid drugs persist (red box). First, the development of new drugs is expensive and time-consuming. In addition, NPs may cause adverse immune effects, such as an inflammatory cytokine storm. Moreover, cost and regulatory challenges exist. In addition, insufficient organ and cell selectivity reduce the effectiveness of small nucleic acid drugs. Many ideas have been proposed to solve these barriers as future directions for small nucleic acid drugs (green boxes). The first is the application of artificial intelligence (AI) in drug discovery. The second is improving the safety profile, which should focus on both the delivery vector and the sequence of small nucleic acids. The third is surface modifications of nucleic acid drugs for delivery to specific cell types. Finally, vectors are screened to deliver nucleic acid drugs more efficiently