The therapeutic potential of RNA interference |
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Authors: | Uprichard Susan L |
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Affiliation: | Department of Molecular and Experimental Medicine, The Scripps Research Institute, 10550 N. Torrey Pines Road, SBR10, La Jolla, CA 92037, USA. sluprich@scripps.edu |
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Abstract: | In recent years, we have witnessed the discovery of a new mechanism of gene regulation called RNA interference (RNAi), which has revitalized interest in the development of nucleic acid-based technologies for therapeutic gene suppression. This review focuses on the potential therapeutic use of RNAi, discussing the theoretical advantages of RNAi-based therapeutics over previous technologies as well as the challenges involved in developing RNAi for clinical use. Also reviewed, are the in vivo proof-of principle experiments that provide the preclinical justification for the continued development of RNAi-based therapeutics. |
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Keywords: | Gene suppression Drug development Delivery Safety RNA interference |
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