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Transgene sequences free of CG dinucleotides lead to high level, long-term expression in the lung independent of plasmid backbone design

Abstract:

Non-viral aerosol gene therapy offers great potential for treating chronic lung diseases of the airways such as cystic fibrosis (CF). Early clinical trials showed that transgene expression in the airways was transient whereas maximal duration of transgene expression is essential in order to minimise the frequency of aerosol treatments. Improved vector design, such as careful selection of the promoter/enhancer, can lead to more persistent levels of transgene expression, but multiple factors af...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Accepted manuscript

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Publisher copy:
10.1016/j.biomaterials.2016.03.029

Authors


Bazzani, RP More by this author
Pringle, IA More by this author
Connolly, MM More by this author
Davies, LA More by this author
Sumner-Jones, SG More by this author
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Publisher:
Elsevier Publisher's website
Journal:
Biomaterials
Volume:
93
Pages:
20-26
Publication date:
2016-03-22
Acceptance date:
2016-03-17
DOI:
ISSN:
1878-5905 and 0142-9612
Pubs id:
pubs:617066
URN:
uri:91be5234-47c6-437f-9f04-cbafbe0704b9
UUID:
91be5234-47c6-437f-9f04-cbafbe0704b9
Local pid:
pubs:617066

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