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Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector

Abstract:

Novel gene-based therapies for disease will depend in many cases on long-term persistent transgene expression. To develop gene therapy strategies for Friedreich's ataxia (FRDA), we have examined the persistence of transgene expression in the brain in vivo provided by the entire 135 kb FXN genomic DNA locus delivered as an infectious bacterial artificial chromosome (iBAC) herpes simplex virus type 1 (HSV-1)-based vector injected in the adult mouse cerebellum. We constructed genomic DNA-reporte...

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Publisher copy:
10.1038/gt.2011.45

Authors


Gimenez-Cassina, A More by this author
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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Gomez-Sebastian, S More by this author
Corona, J-C More by this author
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Journal:
Gene Therapy
Volume:
18
Issue:
10
Pages:
1015-1019
Publication date:
2011-10-05
DOI:
EISSN:
1476-5462
ISSN:
0969-7128
URN:
uuid:66e20079-73a0-4350-9110-2bf47b38a0a8
Source identifiers:
193027
Local pid:
pubs:193027

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