Gene therapy progress and prospects. Downregulating gene expression: the impact of RNA interference

Gene Ther. 2004 Aug;11(16):1241-8. doi: 10.1038/


The control and maintenance of gene expression is critical for cell development and differentiation. Over the last 2 years, our understanding of the role of RNA as a regulator of gene expression has significantly increased. Small RNA molecules are key elements of a machinery that trigger chromosomal modifications, post-transcriptional gene silencing and protein translational blockade depending on the source, the RNA and the nature of the interaction with the target nucleic acid. Currently, the best characterized of this group of RNA-mediated gene regulation pathways is the post-transcriptional gene silencing mechanism known as RNA interference. RNAi is triggered by double-stranded RNA (dsRNA), which induces the formation of a ribonucleoprotein complex that mediates sequence-specific cleavage of the transcript cognate with the input dsRNA. RNAi has been adapted as a functional genomics tool and it has potential as a therapeutic approach. This review will summarize our current understanding of the RNAi mechanism and the various applications of RNAi-based technologies.

Publication types

  • Review

MeSH terms

  • Animals
  • Down-Regulation / genetics
  • Gene Expression Regulation / genetics*
  • Genetic Therapy / trends*
  • Genetic Vectors / genetics
  • Genomics / methods
  • Humans
  • Mammals / genetics
  • RNA Interference / physiology*
  • RNA, Double-Stranded / genetics
  • RNA, Small Interfering / genetics


  • RNA, Double-Stranded
  • RNA, Small Interfering