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Pharmaceuticals 2013, 6(3), 287-294; doi:10.3390/ph6030287

RNAi Therapeutics in Autoimmune Disease

Department of Science and Mathematics, Cedarville University, 251 North Main Street, Cedarville, OH 45314, USA
Department of Oral and Maxillofacial Diagnostic Sciences, University of Florida College of Dentistry, 1600 SW Archer Rd, Gainesville, FL 32610, USA
Author to whom correspondence should be addressed.
Received: 31 December 2012 / Revised: 18 February 2013 / Accepted: 27 February 2013 / Published: 5 March 2013
(This article belongs to the Special Issue RNAi-Based Therapeutics)
View Full-Text   |   Download PDF [127 KB, uploaded 5 March 2013]


Since the discovery of RNA interference (RNAi), excitement has grown over its potential therapeutic uses. Targeting RNAi pathways provides a powerful tool to change biological processes post-transcriptionally in various health conditions such as cancer or autoimmune diseases. Optimum design of shRNA, siRNA, and miRNA enhances stability and specificity of RNAi-based approaches whereas it has to reduce or prevent undesirable immune responses or off-target effects. Recent advances in understanding pathogenesis of autoimmune diseases have allowed application of these tools in vitro as well as in vivo with some degree of success. Further research on the design and delivery of effectors of RNAi pathway and underlying molecular basis of RNAi would warrant practical use of RNAi-based therapeutics in human applications. This review will focus on the approaches used for current therapeutics and their applications in autoimmune diseases, including rheumatoid arthritis and Sjögren’s syndrome. View Full-Text
Keywords: RNA interference; therapeutics; microRNA; small interfering RNA; autoimmune disease RNA interference; therapeutics; microRNA; small interfering RNA; autoimmune disease
This is an open access article distributed under the Creative Commons Attribution License (CC BY 3.0).

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Pauley, K.M.; Cha, S. RNAi Therapeutics in Autoimmune Disease. Pharmaceuticals 2013, 6, 287-294.

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