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RNA interference as potential therapy for neurodegenerative disease

Applications to inclusion-body myositis?

From the Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, IA.

Address correspondence and reprint requests to Dr. Henry Paulson, Department of Neurology, University of Iowa Carver College of Medicine, Iowa City, IA 52242; e-mail: henry-paulson{at}uiowa.edu

The discovery of RNA interference (RNAi) has led to powerful new approaches to silence targeted genes in a sequence-specific manner. The potential therapeutic application of RNAi to neurologic disease is highlighted by the recent success of several laboratories in suppressing the expression of neurodegenerative disease genes in transgenic mouse models. Here I discuss potential applications of RNAi to inclusion-body myositis (IBM) after first reviewing its application more generally to neurologic disease. The clearest application of RNAi to IBM is as a research tool to identify critical target genes that contribute to pathogenesis. Provided that proximal pathogenic targets are identified, RNAi could surface as a potential therapeutic strategy to modulate their expression.

This article was previously published in electronic format as an Expedited E-Pub at www.neurology.org.

Supported by grants from the National Institutes of Health (National Institute of Neurologic Disorders and Stroke) and a Beeson Scholarship from the American Federation of Aging Research.

Disclosure: H.P. is a scientific consultant and serves on the clinical advisory board for Sirna Therapeutics. The author reports no conflicts of interest.

Received June 22, 2005. Accepted in final form October 14, 2005.

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				W. K. Engel and V. Askanas Inclusion-body myositis: Clinical, diagnostic, and pathologic aspects Neurology, January 24, 2006; 66(1_suppl_1): S20 - S29. [Abstract] [Full Text] [PDF]