siRNA therapeutics: big potential from small RNAs
- 21 October 2004
- journal article
- review article
- Published by Springer Nature in Gene Therapy
- Vol. 12 (1) , 5-11
- https://doi.org/10.1038/sj.gt.3302356
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.Keywords
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