Synthetic RNA Therapeutics in Cancer

J Pharmacol Exp Ther. 2023 Aug;386(2):212-223. doi: 10.1124/jpet.123.001587. Epub 2023 May 15.


Recent advances in the RNA delivery system have facilitated the development of a separate field of RNA therapeutics, with modalities including mRNA, microRNA (miRNA), antisense oligonucleotide (ASO), small interfering RNA, and circular (circRNA) that have been incorporated into oncology research. The main advantages of the RNA-based modalities are high flexibility in designing RNA and rapid production for clinical screening. It is challenging to eliminate tumors by tackling a single target in cancer. In the era of precision medicine, RNA-based therapeutic approaches potentially constitute suitable platforms for targeting heterogeneous tumors that possess multiple sub-clonal cancer cell populations. In this review, we discussed how synthetic coding and non-coding RNAs, such as mRNA, miRNA, ASO, and circRNA, can be applied in the development of therapeutics. SIGNIFICANCE STATEMENT: With development of vaccines against coronavirus, RNA-based therapeutics have received attention. Here, the authors discuss different types of RNA-based therapeutics potentially effective against tumor that are highly heterogeneous giving rise to resistance and relapses to the conventional therapeutics. Moreover, this study summarized recent findings suggesting combination approaches of RNA therapeutics and cancer immunotherapy.

Publication types

  • Review

MeSH terms

  • Humans
  • MicroRNAs*
  • Neoplasms* / drug therapy
  • Neoplasms* / therapy
  • Oligonucleotides, Antisense / genetics
  • Oligonucleotides, Antisense / therapeutic use
  • RNA / genetics
  • RNA, Circular / genetics
  • RNA, Circular / therapeutic use
  • RNA, Messenger
  • RNA, Small Interfering / therapeutic use


  • RNA
  • RNA, Circular
  • MicroRNAs
  • RNA, Small Interfering
  • Oligonucleotides, Antisense
  • RNA, Messenger