RNA interference as potential therapy for neurodegenerative disease: applications to inclusion-body myositis?
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.
Paulson
Henry
H
Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA. henry-paulson@uiowa.edu
D000818:Animals; D004195:Disease Models, Animal; D018390:Gene Targeting / Q000379:methods*; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D035683:MicroRNAs / Q000235:genetics / Q000627:therapeutic use*; D018979:Myositis, Inclusion Body / Q000628:therapy*; D012313:RNA / Q000235:genetics; D034622:RNA Interference; D012333:RNA, Messenger / Q000037:antagonists & inhibitors; D034741:RNA, Small Interfering / Q000235:genetics / Q000627:therapeutic use*; D020754:Spinocerebellar Ataxias / Q000235:genetics / Q000628:therapy
D035683:MicroRNAs; D012333:RNA, Messenger; D034741:RNA, Small Interfering; D012313:RNA