RNA interference: from basic research to therapeutic applications

Angew Chem Int Ed Engl. 2009;48(8):1378-98. doi: 10.1002/anie.200802092.

Abstract

An efficient mechanism for the sequence-specific inhibition of gene expression is RNA interference. In this process, double-stranded RNA molecules induce cleavage of a selected target RNA (see picture). This technique has in recent years developed into a standard method of molecular biology. Successful applications in animal models have already led to the initiation of RNAi-based clinical trials as a new therapeutic option.Only ten years ago Andrew Fire and Craig Mello were able to show that double-stranded RNA molecules could inhibit the expression of homologous genes in eukaryotes. This process, termed RNA interference, has developed into a standard method of molecular biology. This Review provides an overview of the molecular processes involved, with a particular focus on the posttranscriptional inhibition of gene expression in mammalian cells, the possible applications in research, and the results of the first clinical studies.

Publication types

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

MeSH terms

  • Animals
  • Drug Delivery Systems
  • Gene Expression Regulation / drug effects
  • Humans
  • MicroRNAs / genetics
  • MicroRNAs / metabolism
  • Neoplasms / genetics
  • Neoplasms / therapy
  • RNA Interference*
  • RNA, Double-Stranded / metabolism
  • RNA, Small Interfering / chemistry*
  • Virus Diseases / therapy

Substances

  • MicroRNAs
  • RNA, Double-Stranded
  • RNA, Small Interfering