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Journal article

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

Abstract:
Background: Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis. Methods: We did this randomised, double-blind, placebo-controlled, phase 2b trial in two cystic fibrosis centres with patients recruited from 18 sites in the UK. Patients (aged ≥12 years) with a fo... Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1016/s2213-2600(15)00245-3

Authors


Armstrong, DK More by this author
Bayfield, KJ More by this author
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Cystic Fibrosis Trust More from this funder
Medicor Foundation More from this funder
National Institute for Health Research More from this funder
Just Gene Therapy More from this funder
Medical Research Council More from this funder
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Publisher:
Elsevier Publisher's website
Journal:
The Lancet. Respiratory medicine Journal website
Volume:
3
Issue:
9
Pages:
684-691
Publication date:
2015-09-05
DOI:
EISSN:
2213-2619
ISSN:
2213-2600
URN:
uuid:f6c88c23-ee48-472a-97fa-e64553ffe2f3
Source identifiers:
531413
Local pid:
pubs:531413
Language:
English
Keywords:

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