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CpG methylation of a plasmid vector results in extended transgene product expression by circumventing induction of immune responses.

Abstract:

Gene therapy has the potential to cure inherited diseases if the delivered genes achieve long-term expression at therapeutic levels in the targeted tissues. Expression is commonly short-lived due to induction of cell-mediated immune responses to the gene therapy vehicle and/or the transgene product, which can be perceived as "foreign" by the host's immune system. Plasmid expression vectors have been used to deliver genes. Bacterial DNA carries immunostimulatory sequences in the form of unmeth...

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Publisher copy:
10.1016/j.ymthe.2003.11.008

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Institution:
University of Oxford
Department:
Oxford, MSD, Clinical Medicine
Journal:
Molecular therapy : the journal of the American Society of Gene Therapy
Volume:
9
Issue:
2
Pages:
249-261
Publication date:
2004-02-05
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
URN:
uuid:441e5c9e-7067-4491-ace7-0683cc1c88d9
Source identifiers:
264555
Local pid:
pubs:264555

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