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Human Molecular Genetics Advance Access published online on August 19, 2003
Human Molecular Genetics, doi:10.1093/hmg/ddg275
© 2003 by Oxford University Press
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©2003 Oxford University Press

Article

THERAPEUTIC GENE SILENCING IN THE NERVOUS SYSTEM

Matthew JA Wood 1*, Barbara Trülzsch 1, Amr Abdelgany 2, and David Beeson 2

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

* To whom correspondence should be addressed. E-mail: matthew.wood{at}anat.ox.ac.uk.


  Abstract

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.


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