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

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 po...

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Main Authors: 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, UK Cystic Fibrosis Gene Therapy Consortium
Format: Journal article
Language:English
Published: NIHR Journals Library 2016