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      The promises and pitfalls of RNA-interference-based therapeutics.

      Nature

      Animals, therapeutic use, genetics, adverse effects, administration & dosage, RNA, Small Interfering, RNA Interference, Humans, trends, Genetic Therapy, Gene Knockdown Techniques, Clinical Trials as Topic

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          Abstract

          The discovery that gene expression can be controlled by the Watson-Crick base-pairing of small RNAs with messenger RNAs containing complementary sequence - a process known as RNA interference - has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA-interference-based therapeutics are already publicly traded.

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          Journal
          19158789
          10.1038/nature07758
          2702667

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