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Human Molecular Genetics 2004 13(Review Issue 2):R275-R288; doi:10.1093/hmg/ddh224
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Human Molecular Genetics, Vol. 13, Review Issue 2 © Oxford University Press 2004; all rights reserved

RNA interference: from model organisms towards therapy for neural and neuromuscular disorders

Steven D. Buckingham1, Behrooz Esmaeili1, Matthew Wood2 and David B. Sattelle1,*

1MRC Functional Genetics Unit and 2Department of Human Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK

Received June 15, 2004; Accepted July 19, 2004

Experimental RNA interference (RNAi) leading to the selective knockdown of gene function is induced by introducing into cells either double stranded RNA (dsRNA), or short interfering RNA (siRNA) fragments into which dsRNA is cut. The siRNA triggers degradation of homologous messenger RNA (mRNA). Widely used as a research tool in the genetic model organisms Caenorhabditis elegans, Drosophila melanogaster and mouse to investigate the function of individual genes, RNAi has also been deployed in genome-wide, specific gene-knockdown screens. Recent rapid progress in the application of RNAi to mammalian cells, including neurons and muscle cells, offers new approaches to drug target identification and validation. Advances in targeted delivery of RNAi-inducing molecules has raised the possibility of using RNAi directly as a therapy for a variety of human genetic and other neural and neuromuscular disorders. Here, we review examples of the application of RNAi to worm, fly and mouse models of such diseases aimed at understanding their pathophysiology and we address problems to be solved in developing RNAi-based therapies.

* To whom correspondence should be addressed. Tel: + 44 1865272145; Fax: + 44 1865282651; Email: david.sattelle{at}anat.ox.ac.uk


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