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RNA Therapeutics Enter Clinical Trials
With human testing now underway, it's time for RNAi therapeutics to silence or shut up
Email: Amy Adams - aadams@the-scientist.com The Scientist 2005, 19(1):28
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Traditional gene therapy is built on a simple premise: If the absence of a gene product causes disease, then adding the missing gene will cure it. But recently some researchers have turned that idea upside down, using gene therapy to silence genes gone bad. The approach takes advantage of a technique called RNA interference (RNAi) to specifically destroy a targeted mRNA and thereby eliminate the resulting protein.
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