Background: RNA is increasingly recognized as a powerful molecule that can be used to
control gene expression. Sophisticated, well-engineered RNA-based regulators are being developed as
Methods: In particular, small activating RNAs (saRNAs) are promising therapeutic options for targeting
human diseases. Numerous saRNAs targeting multiple cancers have been developed in preclinical
models. One saRNA targeting C/EBPα is currently undergoing clinical trials in liver cancer.
Results and Conclusion: In this review, we describe the current working model of the intracellular
mechanism of saRNA, discuss the recent progress of saRNA therapeutics in preclinical and clinical trials,
and current advances in targeted delivery using aptamers in detail.