Despite recent concerns, new research shows that small interfering RNAs (siRNAs) can be effective and safe tools for silencing genes in vivo. The work, published online this week in Nature, demonstrates the use of siRNAs in mouse and hamster, without any demonstrable effect on microRNAs.
The in vivo application of RNA interference for basic research as well as development of therapeutics is rapidly expanding. However, recent work has identified potential for toxic effects in the mouse liver caused by saturation of the microRNA biosynthetic pathway.
David Bumcrot and colleagues show near-complete silencing of two mouse and hamster liver genes by intravenous administration of siRNAs. This silencing is specific to the targeted gene and is not associated with any overt toxicity. They argue that their findings support continued siRNA research and their further development as a new class of therapeutics.
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