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RNAI Therapeutics: A Two-Year Update

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Science  16 Oct 2009:
Vol. 326, Issue 5951, pp. 454
DOI: 10.1126/science.326_454


Two years is a long time in the world of RNAi therapeutics. Since Science last covered the topic in 2007, the first Phase 3 human clinical trial of an siRNA drug has been prematurely terminated; new off-target effects have been identified; and the first miRNA-based therapeutic entered clinical trials. Yet much remains the same, especially the biggest challenge: delivery. As researchers struggle to overcome these obstacles, optimism persists undiminished.