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

Autor: Alton EWFW; Imperial College London, London, UK. Electronic address: e.alton@imperial.ac.uk., Armstrong DK; Royal Hospital for Sick Children, Edinburgh, UK., Ashby D; Imperial College London, London, UK., Bayfield KJ; Imperial College London, London, UK., Bilton D; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Bloomfield EV; Imperial College London, London, UK., Boyd AC; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Brand J; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Buchan R; Western General Hospital, Edinburgh, UK., Calcedo R; Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA., Carvelli P; Imperial College London, London, UK., Chan M; Imperial College London, London, UK., Cheng SH; Genzyme, a Sanofi Company, Framingham, MA, USA., Collie DDS; The Roslin Institute and R(D)SVS, University of Edinburgh, Edinburgh, UK., Cunningham S; Royal Hospital for Sick Children, Edinburgh, UK., Davidson HE; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Davies G; Imperial College London, London, UK., Davies JC; Imperial College London, London, UK., Davies LA; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Dewar MH; Western General Hospital, Edinburgh, UK., Doherty A; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Donovan J; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Dwyer NS; Imperial College London, London, UK., Elgmati HI; Western General Hospital, Edinburgh, UK., Featherstone RF; Imperial College London, London, UK., Gavino J; Imperial College London, London, UK., Gea-Sorli S; Imperial College London, London, UK., Geddes DM; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Gibson JSR; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Gill DR; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Greening AP; Western General Hospital, Edinburgh, UK., Griesenbach U; Imperial College London, London, UK., Hansell DM; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Harman K; Imperial College London, London, UK., Higgins TE; Imperial College London, London, UK., Hodges SL; Imperial College London, London, UK., Hyde SC; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Hyndman L; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Innes JA; Western General Hospital, Edinburgh, UK., Jacob J; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Jones N; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Keogh BF; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Limberis MP; Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA., Lloyd-Evans P; NHS Blood and Transplant, Bristol, UK., Maclean AW; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Manvell MC; Imperial College London, London, UK., McCormick D; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., McGovern M; Western General Hospital, Edinburgh, UK., McLachlan G; The Roslin Institute and R(D)SVS, University of Edinburgh, Edinburgh, UK., Meng C; Imperial College London, London, UK., Montero MA; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Milligan H; Western General Hospital, Edinburgh, UK., Moyce LJ; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Murray GD; Usher Institute of Population Health Sciences and Informatics and Centre for Population Health Sciences, University of Edinburgh, Edinburgh, UK., Nicholson AG; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Osadolor T; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Parra-Leiton J; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Porteous DJ; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Pringle IA; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Punch EK; Imperial College London, London, UK., Pytel KM; Imperial College London, London, UK., Quittner AL; University of Miami, Miami, FL, USA., Rivellini G; Imperial College London, London, UK., Saunders CJ; Imperial College London, London, UK., Scheule RK; Genzyme, a Sanofi Company, Framingham, MA, USA., Sheard S; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Simmonds NJ; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Smith K; NHS Blood and Transplant, Bristol, UK., Smith SN; Imperial College London, London, UK., Soussi N; Imperial College London, London, UK., Soussi S; Imperial College London, London, UK., Spearing EJ; Imperial College London, London, UK., Stevenson BJ; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Sumner-Jones SG; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Turkkila M; Imperial College London, London, UK., Ureta RP; Imperial College London, London, UK., Waller MD; Imperial College London, London, UK., Wasowicz MY; Imperial College London, London, UK., Wilson JM; Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wolstenholme-Hogg P; Genzyme, a Sanofi Company, Framingham, MA, USA.

Publikováno v: The Lancet. Respiratory medicine [Lancet Respir Med] 2015 Sep; Vol. 3 (9), pp. 684-691. Date of Electronic Publication: 2015 Jul 03.