The emerging role of RNA interference in the design of novel therapeutics in oncology

Cell Cycle. 2004 Nov;3(11):1398-401. doi: 10.4161/cc.3.11.1240. Epub 2004 Nov 15.


RNA interference (RNAi) has emerged as the tool of choice for studying gene function. Dubbed the "breakthrough of the year" in 2002 by the journal Science, RNAi is a naturally occurring host defense mechanism that mediates the sequence-specific degradation of target mRNA transcripts and their protein products. The specificity of RNAi makes it an ideal tool for targeted therapeutics against unique fusion oncogene sequences. RNAi may also be effective against viral-mediated oncogenesis and has the potential to enhance tumor sensitivities to existing chemotherapy. The current interest in the success of RNAi-based therapies will depend on the delivery systems that protect the silencing apparatus from endogenous nucleases, sustain tissue-specific expression of the small-interfering RNAs, and prevent the activation of a destructive nonspecific host immune response.

MeSH terms

  • ATP Binding Cassette Transporter, Subfamily B / genetics
  • ATP Binding Cassette Transporter, Subfamily B / metabolism
  • Drug Resistance, Neoplasm / genetics
  • Fusion Proteins, bcr-abl / genetics
  • Fusion Proteins, bcr-abl / metabolism*
  • Gene Products, tat / genetics
  • Gene Products, tat / metabolism*
  • Gene Transfer Techniques
  • Genetic Therapy / methods
  • HIV Infections / genetics
  • HIV Infections / metabolism*
  • HIV Infections / therapy
  • HIV-1 / physiology*
  • Leukemia / genetics
  • Leukemia / metabolism*
  • Leukemia / therapy
  • RNA Interference*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism*
  • Virus Replication / genetics
  • tat Gene Products, Human Immunodeficiency Virus


  • ATP Binding Cassette Transporter, Subfamily B
  • Gene Products, tat
  • RNA, Small Interfering
  • tat Gene Products, Human Immunodeficiency Virus
  • Fusion Proteins, bcr-abl