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Journal article

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Abstract:

Cystic fibrosis (CF) is a chronic, life-limiting disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene leading to abnormal airway surface ion transport, chronic lung infections, inflammation and eventual respiratory failure. With the exception of the small-molecule potentiator, ivacaftor (Kalydeco®, Vertex Pharmaceuticals, Boston, MA, USA), which is suitable for a small proportion of patients, there are no licensed therapies targeting the basic defect. The UK C...

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Publication status:
Published
Peer review status:
Reviewed (other)

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Publisher copy:
10.3310/eme03050

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Publisher:
NIHR Journals Library Publisher's website
Journal:
Efficacy and Mechanism Evaluation Journal website
Volume:
3
Issue:
5
Pages:
1-240
Publication date:
2016-07-22
Acceptance date:
2016-07-19
DOI:
EISSN:
2050-4373
ISSN:
2050-4365
Pmid:
27441329
Source identifiers:
957820
Language:
English
Keywords:
Pubs id:
pubs:957820
UUID:
uuid:1d452da7-a1b1-4d93-ae93-e22c76576751
Local pid:
pubs:957820
Deposit date:
2019-06-18

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