Current prospects and challenges for epilepsy gene therapy

This review addresses the state of gene therapy research for the treatment of epilepsy. Preclinical studies have demonstrated the anti-seizure efficacy of viral vector-based gene transfer through the use of a variety of strategies — from modulating classic neurotransmitter systems to targeting or ov...

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Bibliographic Details
Published in:Experimental neurology 2013-06, Vol.244, p.27-35
Main Authors: Weinberg, Marc S., McCown, Thomas J.
Format: Article
Language:English
Subjects:
Adeno-associated virus
Adenosine
Animals
Epilepsy
Epilepsy - genetics
Epilepsy - therapy
Galanin
Gene Transfer Techniques
Genetic Therapy - methods
Genetic Vectors - physiology
Humans
Immunity
NPY
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Summary:This review addresses the state of gene therapy research for the treatment of epilepsy. Preclinical studies have demonstrated the anti-seizure efficacy of viral vector-based gene transfer through the use of a variety of strategies — from modulating classic neurotransmitter systems to targeting or overexpressing of neuropeptide receptors in seizure-specific brain regions. While these studies provide substantive proof of principle for viral vector gene therapy, future studies must address the challenges of vector immunity, cellular specificity and effective global delivery. As these issues are resolved, viral vector gene therapy should significantly impact the treatment of intractable epilepsy.
ISSN:0014-4886
1090-2430
DOI:10.1016/j.expneurol.2011.10.003