RNA-targeted therapeutics in cancer clinical trials: Current status and future directions

•Substantial development in creating and validating RNA therapeutics has been made.•There are more than 100 cancer clinical trials reported, so far.•Challenges related with delivery, targeting and off-target effects are being optimized.•The success of these strategies in the treatment of cancer is still to come.

Recent advances in RNA delivery and target selection provide unprecedented opportunities for cancer treatment, especially for cancers that are particularly hard to treat with existing drugs. Small interfering RNAs, microRNAs, and antisense oligonucleotides are the most widely used strategies for silencing gene expression. In this review, we summarize how these approaches were used to develop drugs targeting RNA in human cells. Then, we review the current state of clinical trials of these agents for different types of cancer and outcomes from published data. Finally, we discuss lessons learned from completed studies and future directions for this class of drugs.