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

@inproceedings{Alton2015RepeatedNO,
  title={Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial},
  author={Eric W.F.W. Alton and David K Armstrong and Deborah Ashby and Katie J Bayfield and D Bilton and Emily V Bloomfield and A Christopher Boyd and June Brand and Ruaridh Buchan and Roberto Calcedo and Paula Carvelli and Mario Lapizco Chan and Shuk Han Cheng and David S Collie and Steve Cunningham and Heather Elin Davidson and Gwyneth Davies and Jane C Davies and Lee Adrian Davies and Michael L. Dewar and Ann Alderson Doherty and Jackie Donovan and Natalie S Dwyer and Hala I Elgmati and Rosanna F. Featherstone and Jemyr Gavino and Sabrina Gea-Sorli and Duncan M. Geddes and James Randall Gibson and Deborah Rebecca Gill and Andrew Peter Greening and Uta Griesenbach and David M. Hansell and Katharine Harman and Tracy E. Higgins and Samantha L Hodges and Stephen Charles Hyde and Laura Hyndman and J. Alastair Innes and Joseph Jacob and Nancy Wright Jones and Brian F. Keogh and Maria P. Limberis and Paul Lloyd-Evans and Alan W Maclean and Michelle C Manvell and Dominique McCormick and Michael H. McGovern and Gerry McLachlan and Cuixiang Meng and Maria A Montero and Hazel Milligan and Laura J Moyce and Graham K. Murray and Andrew G. Nicholson and Tina Osadolor and Javier Parra-Leiton and David J. Porteous and Ian A. Pringle and Emma Kathleen Punch and Kamila Malgorzata Pytel and Alexandra L. L Quittner and Gina Rivellini and Clare J Saunders and Ronald K. Scheule and Sarah Sheard and Nicholas Simmonds and Keith Marion Smith and Stephen Neal Smith and Najwa Soussi and Samia Soussi and Emma J Spearing and Barbara J Stevenson and Stephanie G. Sumner-Jones and Minna Turkkila and Rosa P Ureta and Michael David Waller and Marguerite Y Wasowicz and James Meredith Wilson and Paul C Wolstenholme-Hogg},
  booktitle={The Lancet. Respiratory medicine},
  year={2015}
}
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 forced expiratory volume in 1 s (FEV1) of… CONTINUE READING
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This study was funded by the Effi cacy and Mechanism Evaluation (EME) Programme, a Medical Research Council and NIHR Articles www.thelancet.com/respiratory Published online July 3

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