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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.

Original publication

DOI

10.1093/hmg/ddg275

Type

Journal article

Journal

Hum Mol Genet

Publication Date

15/10/2003

Volume

12 Spec No 2

Pages

R279 - R284

Keywords

Gene Silencing, Humans, Nervous System, RNA Interference