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CpG-free plasmid expression cassettes for cystic fibrosis gene therapy

Abstract:

Clinical studies are underway for the aerosol delivery of plasmid DNA complexed with Genzyme Lipid GL67A to the lungs of patients with cystic fibrosis (CF). Plasmid vectors contain several functional elements all of which play a role in determining the efficacy of the final clinical product. To optimise the final plasmid, variations of CpG-free 5' enhancer elements and 3'UTR regions were inserted into a common CpG-free, plasmid backbone containing Luciferase or CFTR transgenes. Plasmids were ...

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Journal:
Biomaterials
Volume:
33
Issue:
28
Pages:
6833-6842
Publication date:
2012-10-01
DOI:
EISSN:
1878-5905
ISSN:
0142-9612
Source identifiers:
344771
Language:
English
Keywords:
Pubs id:
pubs:344771
UUID:
uuid:1d2fd7d2-700c-45e4-bca3-664532f3eba3
Local pid:
pubs:344771
Deposit date:
2012-12-19

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