Therapeutic gene silencing in neurological disorders, using interfering RNA

The development of interfering RNA (RNAi) from a naturally occurring phenomenon to a tool for mediating highly specific gene silencing provides an exciting prospect as a novel therapeutic strategy for a wide range of disorders. Although the efficacy of RNAi as a research tool for analysing gene func...

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Bibliographic Details
Published in:Journal of molecular medicine (Berlin, Germany) Germany), 2005-06, Vol.83 (6), p.413-419
Main Authors: RALPH, G. Scott, MAZARAKIS, Nicholas D, AZZOUZ, Mimoun
Format: Article
Language:English
Subjects:
Amyotrophic Lateral Sclerosis - therapy
Biological and medical sciences
Gene Silencing
General aspects
Genetic Therapy - methods
Humans
Medical sciences
Nervous System Diseases - therapy
Neurological disorders
RNA, Messenger - metabolism
RNA, Small Interfering - therapeutic use
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Summary:The development of interfering RNA (RNAi) from a naturally occurring phenomenon to a tool for mediating highly specific gene silencing provides an exciting prospect as a novel therapeutic strategy for a wide range of disorders. Although the efficacy of RNAi as a research tool for analysing gene function has been well demonstrated in several cell types, the therapeutic potential of RNAi-mediated gene silencing has only recently started to be investigated. Several neurodegenerative disorders provide particularly suitable candidates for RNAi based therapy; however, many hurdles preclude the success of therapeutic application. These include the challenge of delivering active RNAi molecules to the specific target cell populations where they are required and appropriate regulation of gene suppression, such as to maintain a long-lasting therapeutic effect. Furthermore, for safety reasons, off-target effects should be minimised. Here we review the advancement of RNAi technology for therapeutic application and highlight the potential of targeted gene silencing for the treatment of neurodegenerative diseases.
ISSN:0946-2716
1432-1440
DOI:10.1007/s00109-005-0649-1