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RNA interference and potential therapeutic applications of short interfering RNAs

Cancer Gene Therapy volume 12, pages 787–795 (2005)Cite this article

Abstract

RNA interference is an endogenous gene-silencing mechanism that involves double-stranded RNA-mediated sequence-specific mRNA degradation. The discovery of this pathway together with the elucidation of the structure and function of short interfering RNAs — the effector molecules of RNA interference — has had an enormous impact on experimental biology. RNA interference technologies are currently the most widely utilized techniques in functional genomic studies. Furthermore, there is an intense research effort aimed at developing short interfering RNAs for therapeutic purposes. A number of proof-of-principle experiments have demonstrated the clinical potential of appropriately designed short interfering RNAs in various diseases including viral infections, cancer and neurodegenerative disorders. Already, in such a short time from their discovery, Acuity Pharmaceuticals (August 2004) and Sirna Therapeutics (September 2004) have filed Investigational New Drug applications with the US FDA to begin clinical trials with modified siRNA molecules in patients with age-related macular degeneration. This review will give a brief overview of the mechanism of RNA interference and applications of the pathway in experimental biology will be discussed. The article will focus on recent developments related to the use of RNA interference technologies in mammalian systems and on potential clinical applications of short interfering RNA-mediated RNA interference.

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Acknowledgements

The support of the Australian Institute of Nuclear Science and Engineering is acknowledged. TCK was the recipient of AINSE awards. The Molecular Radiation Biology Laboratory is also supported by the National Health and Medical Research Council of Australia (Grant no. 209125).

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  1. Molecular Radiation Biology, Trescowthick Research Laboratories, Peter MacCallum Cancer Centre, Melbourne, Australia

    Tom C Karagiannis

  2. Epigenetics in Human Health and Disease, Baker Medical Research Institute, The Alfred Medical Research and Education Precinct, Prahran, Victoria, Australia

    Assam El-Osta

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Karagiannis, T., El-Osta, A. RNA interference and potential therapeutic applications of short interfering RNAs. Cancer Gene Ther 12, 787–795 (2005). https://doi.org/10.1038/sj.cgt.7700857

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