MicroRNA therapeutics

Gene Ther. 2011 Dec;18(12):1104-10. doi: 10.1038/gt.2011.50. Epub 2011 Apr 28.


MicroRNAs (miRNAs) provide new therapeutic targets for many diseases, while their myriad roles in development and cellular processes make them fascinating to study. We still do not fully understand the molecular mechanisms by which miRNAs regulate gene expression nor do we know the complete repertoire of mRNAs each miRNA regulates. However, recent progress in the development of effective strategies to block miRNAs suggests that anti-miRNA drugs may soon be used in the clinic.

Publication types

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

MeSH terms

  • Argonaute Proteins / pharmacology
  • Gene Expression Regulation
  • Gene Transfer Techniques
  • Genetic Therapy / methods
  • Humans
  • MicroRNAs / antagonists & inhibitors*
  • MicroRNAs / physiology
  • RNA Polymerase II / metabolism
  • RNA, Viral / physiology
  • Ribonuclease III


  • Argonaute Proteins
  • MicroRNAs
  • RNA, Viral
  • RNA Polymerase II
  • Ribonuclease III