RNA interference: a tool for querying nervous system function and an emerging therapy.
RNA interference (RNAi), a mediator of gene silencing, has swiftly become one of the most exciting and applicable biological discoveries. There has been rapid progress in identifying RNAi pathway components and elucidating the mechanisms of microRNA (miRNA) biogenesis and gene suppression. As a result, RNAi technologies have been successfully employed in a variety of systems as biological tools, and studies are underway to test the therapeutic utility of RNAi. In the span of several years, significant advances in the delivery of inhibitory RNAs in the nervous system have been made. We have glimpses into how endogenous miRNAs interface with neuronal development and function; in addition, RNAi has shown therapeutic efficacy in several mouse models of human neurological conditions. In this review, we summarize the current state-of-the-art of RNAi and its utility to neuroscientists.
Davidson
Beverly L
BL
Department of Internal Medicine, University of Iowa, Iowa City, IA 52242, USA. beverly-davidson@uiowa.edu
Boudreau
Ryan L
RL
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008954:Models, Biological; D009422:Nervous System Diseases / Q000628:therapy*; D009424:Nervous System Physiological Phenomena; D034622:RNA Interference / Q000502:physiology*; D034741:RNA, Small Interfering / Q000627:therapeutic use
D034741:RNA, Small Interfering