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Genes 2013, 4(3), 457-484;

Antisense Gene Silencing: Therapy for Neurodegenerative Disorders?

Danish Dementia Research Centre, Neurogenetics Clinic, Department of Neurology, Section 6702, Rigshospitalet, Copenhagen University Hospital, Blegdamsvej 9, DK-2100, Copenhagen Ø, Denmark
Department of Cellular and Molecular Medicine, Section of Neurogenetics, The Panum Institute, University of Copenhagen, Blegdamsvej 3, DK-2200 Copenhagen N, Denmark
Author to whom correspondence should be addressed.
Received: 20 May 2013 / Revised: 11 July 2013 / Accepted: 13 August 2013 / Published: 10 September 2013
(This article belongs to the Special Issue Gene Silencing)
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Since the first reports that double-stranded RNAs can efficiently silence gene expression in C. elegans, the technology of RNA interference (RNAi) has been intensively exploited as an experimental tool to study gene function. With the subsequent discovery that RNAi could also be applied to mammalian cells, the technology of RNAi expanded from being a valuable experimental tool to being an applicable method for gene-specific therapeutic regulation, and much effort has been put into further refinement of the technique. This review will focus on how RNAi has developed over the years and how the technique is exploited in a pre-clinical and clinical perspective in relation to neurodegenerative disorders. View Full-Text
Keywords: RNA interference; neurodegenerative disorders; CNS RNA interference; neurodegenerative disorders; CNS

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This is an open access article distributed under the Creative Commons Attribution License (CC BY 3.0).

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Nielsen, T.T.; Nielsen, J.E. Antisense Gene Silencing: Therapy for Neurodegenerative Disorders? Genes 2013, 4, 457-484.

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