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siRNA therapeutics: big potential from small RNAs

Gene Therapy volume 12, pages 5–11 (2005)Cite this article

Abstract

RNA interference (RNAi) is now an umbrella term referring to post-transcriptional gene silencing mediated by either degradation or translation arrest of target RNA. This process is initiated by double-stranded RNA with sequence homology driving specificity. The discovery that 21–23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. While much work remains to optimize delivery and maintain specificity, the therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise.

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Acknowledgements

This work was supported by the National Institutes of Health (DK035592 and GM62487). RCCR was supported by NIH 5T32 GM0347.

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  1. Department of Biological Sciences, Vanderbilt University, Nashville, TN, USA

    R C C Ryther, A S Flynt & J G Patton

  2. Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, TN, USA

    J A Phillips III

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  1. R C C Ryther
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Ryther, R., Flynt, A., Phillips, J. et al. siRNA therapeutics: big potential from small RNAs. Gene Ther 12, 5–11 (2005). https://doi.org/10.1038/sj.gt.3302356

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