Advantages and disadvantages of RNA therapeutics

Prog Mol Biol Transl Sci. 2024:203:151-164. doi: 10.1016/bs.pmbts.2023.12.016. Epub 2024 Jan 25.

Abstract

RNA therapeutics is an innovative and rapidly evolving field at the forefront of medical research and biotechnology. Recently, many studies have shown that diverse RNA types play important roles in cells. Besides the protein translation coding, they also express and regulate a variety of cellular pathways. Indeed, along with the research and studies, many drugs and vaccines were developed from RNAs, including both coding and non-coding RNA. Some cases were approved to be medicines or under clinical trial. After years of use and application, they have shown a bright opportunity to prevent and treat many fatal and rare diseases with many strong points, such as fast production and long-term effects. Besides, they still have some drawbacks that need to be overcome, like stability or delivery to become the new generation of medicine. Therefore, this chapter focuses on providing an overview of the advantages and disadvantages of RNA therapeutics as well as some crucial points for future development.

Keywords: Off-target; RNA medicine; RNA therapeutics; RNA-based advantage; RNA-based disadvantage; Undruggable-target.

MeSH terms

  • Humans
  • RNA* / genetics
  • RNA* / therapeutic use

Substances

  • RNA