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

W.F.W. Alton, Eric; K Armstrong, David; Ashby, Deborah; J Bayfield, Katie; Bilton, Diana; V Bloomfield, Emily; Christopher Boyd, A.; Brand, June; Buchan, Ruaridh; Calcedo, Roberto; Carvelli, Paula; Chan, Mario; H. Cheng, Seng; Collie, David; Cunningham, Steve; Davidson, Heather; Davies, Gwyneth; C. Davies, Jane; A. Davies, Lee; H Dewar, Maria; Doherty, Ann; Donovan, Jackie; S Dwyer, Natalie; I Elgmati, Hala; F Featherstone, Rosanna; Gavino, Jemyr; Gea‐Sorlí, Sabrina; M. Geddes, Duncan; Sr Gibson, James; R. Gill, Deborah; P Greening, A; Griesenbach, Uta; M. Hansell, David; Harman, Katharine; Higgins, Tracy; L. Hodges, Samantha; C. Hyde, Stephen; Hyndman, Laura; Alastair Innes, J.; Jacob, Joseph; E. Jones, Nancy; F Keogh, Brian; P. Limberis, Maria; Lloyd-Evans, Paul; Maclean, Alan; C Manvell, Michelle; McCormick, Dominique; McGovern, Michael; McLachlan, Gerry; Meng, Cuixiang; Angeles Montero, M; Milligan, Hazel; Moyce, Laura; Murray, Gordon; G. Nicholson, Andrew; Osadolor, Tina; Parra-Leiton, Javier; J. Porteous, David; A. Pringle, Ian; K Punch, Emma; M Pytel, Kamila; L. Quittner, Alexandra; Rivellini, Gina; Saunders, Clare; K. Scheule, Ronald; Sheard, Sarah; J. Simmonds, Nicholas; Smith, Keith; N. Smith, Stephen; Soussi, Najwa; Soussi, Samia; J Spearing, Emma; Stevenson, Barbara; Jones, Stephanie; Turkkila, Minna; P Ureta, Rosa; D Waller, Michael; Y Wasowicz, Marguerite; M. Wilson, James; Wolstenholme-Hogg, Paul

doi:10.1016/s2213-2600(15)00245-3

Public

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

Published in The Lancet Respiratory Medicine • Jul 5, 2015

Authors:

Eric W.F.W. Alton

David K Armstrong

Deborah Ashby

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, doub...

Subject

Medicine

Placebo

Cystic fibrosis

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