title: Therapeutic gene silencing in the nervous system.
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.
authors.affiliation: Department of Human Anatomy and Genetics, Oxford University, Oxford, UK. matthew.wood@anat.ox.ac.uk
authors.forename: Matthew J A
authors.initials: MJ
authors.lastname: Wood
authors.forename: Barbara
authors.initials: B
authors.lastname: Trülzsch
authors.forename: Amr
authors.initials: A
authors.lastname: Abdelgany
authors.forename: David
authors.initials: D
authors.lastname: Beeson
mesh_terms: D020868:Gene Silencing; D006801:Humans; D009420:Nervous System / Q000378:metabolism*; D034622:RNA Interference