RNA interference: potential therapeutic targets

Appl Microbiol Biotechnol. 2004 Nov;65(6):649-57. doi: 10.1007/s00253-004-1732-1. Epub 2004 Sep 15.

Abstract

One of the most exciting findings in recent years has been the discovery of RNA interference (RNAi). RNAi methodologies hold the promise to selectively inhibit gene expression in mammals. RNAi is an innate cellular process activated when a double-stranded RNA (dsRNA) molecule of greater than 19 duplex nucleotides enters the cell, causing the degradation of not only the invading dsRNA molecule, but also single-stranded (ssRNAs) RNAs of identical sequences, including endogenous mRNAs. The use of RNAi for genetic-based therapies has been widely studied, especially in viral infections, cancers, and inherited genetic disorders. As such, RNAi technology is a potentially useful method to develop highly specific dsRNA-based gene-silencing therapeutics.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Silencing
  • Humans
  • Neoplasms / drug therapy
  • Nervous System Diseases / drug therapy
  • RNA Interference*
  • RNA, Small Interfering / therapeutic use*
  • Virus Diseases / drug therapy

Substances

  • RNA, Small Interfering