Gene silencing by siRNA (short interfering RNA) is a still developing field in biology and has evolved as a novel post-transcriptional gene silencing strategy with therapeutic potential. With siRNAs, virtually every gene in the human genome contributing to a disease becomes amenable to regulation, thus opening unprecedented opportunities for drug discovery. Besides the well-established role for siRNA as a tool for target screening and validation in vitro, recent progress of siRNA delivery in vivo raised expectations for siRNA drugs as the up-and-coming ‘magic bullet’. Whether siRNA compounds will make it as novel chemical entities from ‘bench to bedside’ will probably depend largely on improving their pharmacokinetics in terms of plasma stability and cellular uptake. Whereas locally administered siRNAs have already entered the first clinical trials, strategies for successful systemic delivery of siRNA are still in a preclinical stage of development. Irrespective of its therapeutic potential, RNAi (RNA interference) has unambiguously become a valuable tool for basic research in biology and thereby it will continue to have a major impact on medical science. In this review, we will give a brief overview about the history and current understanding of RNAi and focus on potential applications, especially as a therapeutic option to treat human disease.
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Review Article| December 12 2005
Short interfering RNA (siRNA): tool or therapeutic?
1Section of Experimental Oncology/Molecular Pharmacology, Department of Clinical Pharmacology, Medical University Vienna, Währinger Gürtel 18-20, A-1090 Vienna, Austria
Correspondence: Dr Volker Wacheck (email Volker.Wacheck@meduniwien.ac.at).
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Publisher: Portland Press Ltd
Received: May 20 2005
Revision Received: June 30 2005
Accepted: July 05 2005
Online ISSN: 1470-8736
Print ISSN: 0143-5221
The Biochemical Society
Daniel Cejka, Doris Losert, Volker Wacheck; Short interfering RNA (siRNA): tool or therapeutic?. Clin Sci (Lond) 1 January 2006; 110 (1): 47–58. doi: https://doi.org/10.1042/CS20050162
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