RNA therapeutics history and future perspectives

Prog Mol Biol Transl Sci. 2024:203:99-114. doi: 10.1016/bs.pmbts.2024.01.004. Epub 2024 Jan 24.

Abstract

Ribonucleic acid (RNA) therapeutics have significantly used RNA-based drugs to the prevention and treatment of diseases as effective messenger RNA-based vaccines in response to the COVID-19 pandemic. The RNA therapeutics with five classes including antisense oligonucleotide, small interfering RNA, microRNA, APTAMER and messenger RNAs are being quickly developed to treat various human diseases as neurological disease, cardiovascular disease, genetic and rare disease, cancer disease, coronavirus disease… which cannot be treated by other conventional drugs as small molecule-based drugs and antibodies. Therefore, the discovery of these RNA therapeutics created a new avenue for treatment of various human diseases. This chapter demonstrates the history of important discoveries in RNA biology and their impact on key developments in RNA therapeutics as well as the advantages of RNA therapeutics; RNA therapeutics describes the action mechanisms and examples of RNA-based drugs approved for treatment of various disease; and RNA therapeutics discusses delivery methods for RNA therapeutics to target organs and cells. In conclusion, this chapter is designed to offer an updated important development and advance of RNA therapeutics for the prevention and treatment of various human diseases.

Keywords: Future perfectives; History; Human diseases; RNA therapeutics; RNA-based therapeutics.

MeSH terms

  • Humans
  • MicroRNAs* / genetics
  • Oligonucleotides, Antisense / therapeutic use
  • Pandemics
  • RNA*
  • RNA, Messenger
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / therapeutic use

Substances

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