Therapeutic applications of ribozymes and riboswitches

Curr Opin Pharmacol. 2010 Oct;10(5):551-6. doi: 10.1016/j.coph.2010.07.002. Epub 2010 Aug 3.


Therapeutic approaches employing RNA as a tool or as a drug target have recently emerged and have been employed for various applications-ranging from cancer treatment to virus infection. Despite the paucity of its molecular groups compared to proteins, RNA has nevertheless proved to be an excellent choice for researchers who have aspired to develop therapeutic tools. Ribozymes and riboswitches are RNA-based therapeutic tools that are most often employed to knockdown gene expression and to inhibit bacterial infections, respectively. The aim of this review is to summarize recent advances observed in ribozyme- and riboswitch-based therapeutic applications that, in some cases, have reached clinical trials.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Gene Knockdown Techniques
  • Genetic Therapy
  • HIV Infections / genetics
  • HIV Infections / therapy
  • Humans
  • Neoplasms / genetics
  • Neoplasms / therapy
  • Prion Diseases / genetics
  • Prion Diseases / therapy
  • RNA, Catalytic / genetics
  • RNA, Catalytic / therapeutic use*
  • Riboswitch / physiology*


  • RNA, Catalytic
  • Riboswitch
  • hammerhead ribozyme