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The discovery of RNA interference caused a revival of the concept of short nucleic acids application as molecular tools for therapeutic control of gene expression. After almost a decade of intensive research by leading pharmaceutical companies, there is only a limited number of clinical trials of synthetic siRNAs. In this review, we shall summarize the present status of knowledge of RNAi mechanism of action and discuss the potential of the use of siRNA in theraphy. We shall also provide data on recent achievements in current clinical trials based on RNAi techniques.
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Barbara Nawrot, Zaklad Chemii Bioorganicznej, Centrum Badan Molekularnych i Makromolekularnych, Polska Akademia Nauk, ul. Sienkiewicza 112, 90-363 Lodz, Poland