Journal Article

Therapeutic gene silencing in the nervous system

Matthew J.A. Wood, Barbara Trülzsch, Amr Abdelgany and David Beeson

in Human Molecular Genetics

Volume 12, issue suppl_2, pages R279-R284
Published in print October 2003 | ISSN: 0964-6906
Published online October 2003 | e-ISSN: 1460-2083 | DOI:
Therapeutic gene silencing in the nervous system

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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.

Journal Article.  4972 words.  Illustrated.

Subjects: Genetics and Genomics

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