Drugs to treat diseases from cancer to AIDS could soon rely on short strands of RNA for their effects. But scientists must first work out how to navigate these fragments around the body. Nathan Blow reports. The remarkable ability of short sequences of synthetic RNA to interfere with messenger RNA and thereby silence the activity of specific genes has proved incredibly helpful to geneticists wrestling with genetic function. And the push to harness this RNA interference (RNAi) for therapeutic use is now beginning to make headway. In the six years since the first paper reporting RNAi gene silencing in mammals was published, at least six therapeutic programmes based on the concept have moved into clinical trials.
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