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...
Expand abstract
- Publication status:
- Published
- Peer review status:
- Reviewed (other)
Actions
Access Document
- Files:
-
-
(Version of record, pdf, 36.6MB)
-
- Publisher copy:
- 10.3310/eme03050
Why is the content I wish to access not available via ORA?
Bibliographic data (the information relating to research outputs) and full-text items (e.g. articles, theses, reports, etc.) arrive in ORA from several different sources. Unfortunately we are not able to make available the full-text for every research output.
Please contact the ORA team (
) if you have queries regarding unavailable content OR if you are aware of a full-text copy we can make available.
) if you have queries regarding unavailable content OR if you are aware of a full-text copy we can make available.
Content may be unavailable for the following four reasons
-
Version unsuitable
-
We have not obtained a suitable full-text for a given research output. See this page for more information.
-
Recently completed
-
Sometimes content is held in ORA but is unavailable for a fixed period of time to comply with the policies and wishes of rights holders.
-
Permissions
-
All content made available in ORA should comply with relevant rights, such as copyright. See this page for more information.
-
Clearance
-
Some thesis volumes scanned as part of the digitisation scheme funded by Dr Leonard Polonsky are currently unavailable due to sensitive material or uncleared third-party copyright content. We are attempting to contact authors whose theses are affected.
Alternative access to the full-text
You may be able to access the full-text directly from the publisher's website using the 'Publisher Copy' link in the 'Links & Downloads' box from a research output's ORA record page. This method may require an institutional or individual subscription to the journal/resource.
Authors
Funding
Bibliographic Details
- 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
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:957820
- UUID:
-
uuid:1d452da7-a1b1-4d93-ae93-e22c76576751
- Local pid:
- pubs:957820
- Deposit date:
- 2019-06-18
Terms of use
- Copyright holder:
- Queen’s Printer and Controller
- Copyright date:
- 2016
- Notes:
- © Queen’s Printer and Controller of HMSO 2016. This work was produced by Alton et al. under the terms of a commissioning contract issued by the Secretary of State for Health. This issue may be freely reproduced for the purposes of private research and study and extracts (or indeed, the full report) may be included in professional journals provided that suitable acknowledgement is made and the reproduction is not associated with any form of advertising. Applications for commercial reproduction should be addressed to: NIHR Journals Library, National Institute for Health Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK.
If you are the owner of this record, you can report an update to it here: Report update to this record