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Human Molecular Genetics Advance Access originally published online on August 19, 2003
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Human Molecular Genetics, 2003, Vol. 12, Review Issue 2 R279-R284
DOI: 10.1093/hmg/ddg275
© 2003 Oxford University Press

Therapeutic gene silencing in the nervous system

Matthew J.A. Wood1,*, Barbara Trülzsch1, Amr Abdelgany2 and David Beeson2

1Department of Human Anatomy and Genetics, South Parks Road, Oxford University, Oxford OX1 3QX, UK and 2Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, Oxford University, Oxford OX3 9DU, UK

Received July 28, 2003; Accepted August 4, 2003

Progress in the understanding of RNA biology has brought into focus the prospect of using RNA-based therapeutics as a novel approach to treat human disease. In particular, following the discovery of the RNA interference (RNAi) pathway, the emergence of technology based on small interfering RNA (siRNA) now offers a powerful and highly specific tool for therapeutic gene silencing. Many neurological diseases, including neurodegenerative disorders, tumours and retinal disease are likely candidates to benefit from such advances. The challenges ahead will be to identify appropriate disease gene targets and, crucially, to understand the biological parameters that determine safe, precise and effective delivery and function of RNA-based therapeutic molecules within the unique environment of the nervous system.

* To whom correspondence should be addressed. Tel: +44 1865272419; Fax: +44 1865272420; Email: matthew.wood{at}anat.ox.ac.uk


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