Drugging RNAi

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Science  06 Mar 2015:
Vol. 347, Issue 6226, pp. 1069-1070
DOI: 10.1126/science.1252967

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RNA interference (RNAi)–based drugs harness endogenous posttranscriptional gene silencing pathways for therapeutic purposes. The goal is to turn down or shut off the expression of genes known to contribute to or cause disease. RNAi “triggers” are typically double-stranded RNAs (dsRNAs) of which one strand has a sequence complementary to that of a messenger RNA (mRNA), resulting in the reduction or elimination of that an mRNA and its corresponding protein product. The dsRNAs can be provided as synthetic oligonucleotides or as genetic DNA templates from which the RNAi triggers are transcribed in the target cells (vector-based transcriptional RNAi) (see the figure).