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The silent treatment: siRNAs as small molecule drugs

Gene Therapy volume 13, pages 541–552 (2006)Cite this article

Abstract

As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Two strategies can introduce small RNAs into the cytoplasm of cells, where they are active – a drug approach where double-stranded RNAs are administered in complexes designed for intracellular delivery and a gene therapy approach to express precursor RNAs from viral vectors. Phase I clinical studies have already begun to test the therapeutic potential of small RNA drugs that silence disease-related genes by RNAi. This review will discuss progress in developing and testing small RNAi-based drugs and potential obstacles.

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Acknowledgements

This work was supported by NIH grants AI058695 and AI056900. We thank members of the laboratory and our collaborators for many useful suggestions.

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  1. CBR Institute for Biomedical Research and Department of Pediatrics, Harvard Medical School, Boston, MA, USA

    D M Dykxhoorn, D Palliser & J Lieberman

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  1. D M Dykxhoorn
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  2. D Palliser
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  3. J Lieberman
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Correspondence to J Lieberman.

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Dykxhoorn, D., Palliser, D. & Lieberman, J. The silent treatment: siRNAs as small molecule drugs. Gene Ther 13, 541–552 (2006). https://doi.org/10.1038/sj.gt.3302703

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  • DOI: https://doi.org/10.1038/sj.gt.3302703

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