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

Autor: Alton, EWFW, Armstrong, DK, Ashby, D, Bayfield, KJ, Bilton, D, Bloomfield, EV, Boyd, AC, Brand, J, Buchan, R, Calcedo, R, Carvelli, P, Chan, M, Cheng, SH, Collie, DS, Cunningham, S, Davidson, HE, Davies, G, Davies, JC, Davies, LA, Dewar, MH, Doherty, A, Donovan, J, Dwyer, NS, Elgmati, HI, Featherstone, RF, Gavino, J, Gea-Sorli, S, Geddes, DM, Gibson, JSR, Gill, DR, Greening, AP, Griesenbach, U, Hansell, DM, Harman, K, Higgins, TE, Hodges, SL, Hyde, SC, Hyndman, L, Innes, JA, Jacob, J, Jones, N, Keogh, BF, Limberis, MP, Lloyd-Evans, P, Maclean, AW, Manvell, MC, McCormick, D, McGovern, M, McLachlan, G, Meng, C, Montero, MA, Milligan, H, Moyce, LJ, Murray, GD, Nicholson, AG, Osadolor, T, Parra-Leiton, J, Porteous, DJ, Pringle, IA, Punch, EK, Pytel, KM, Quittner, AL, Rivellini, G, Saunders, CJ, Scheule, RK, Sheard, S, Simmonds, NJ, Smith, K, Smith, SN, Soussi, N, Soussi, S, Spearing, EJ, Stevenson, BJ, Sumner-Jones, SG, Turkkila, M, Ureta, RP, Waller, MD, Wasowicz, MY, Wilson, JM, Wolstenholme-Hogg, P, Consortium, UK Cystic Fibrosis Gene Therapy

Publikováno v: Efficacy and Mechanism Evaluation, Vol 3, Iss 5 (2016)

BackgroundCystic 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 resp

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https://doaj.org/article/09b29fe1b155492fba24a177fe4be24c

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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.