Journal article icon

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) wit...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


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

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
RDM
Sub department:
RDM Clinical Laboratory Sciences
Role:
Author
Expand authors...
Cystic Fibrosis Trust More from this funder
National Institute for Health Research More from this funder
Medical Research Council More from this funder
NHS ResearchScotland More from this funder
Just Gene Therapy More from this funder
Expand funders...
Publisher:
Elsevier Publisher's website
Journal:
Lancet Respiratory Medicine Journal website
Volume:
3
Issue:
9
Pages:
684-691
Publication date:
2015-09-03
DOI:
EISSN:
2213-2619
ISSN:
2213-2600
Source identifiers:
531413
Language:
English
Keywords:
Pubs id:
pubs:531413
UUID:
uuid:f6c88c23-ee48-472a-97fa-e64553ffe2f3
Local pid:
pubs:531413
Deposit date:
2015-11-04

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP