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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.

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第一作者: Eric W F W,Alton
第一单位: Imperial College London, London, UK. Electronic address: e.alton@imperial.ac.uk.
作者: Eric W F W,Alton [1] ; David K,Armstrong [2] ; Deborah,Ashby [3] ; Katie J,Bayfield [3] ; Diana,Bilton [4] ; Emily V,Bloomfield [3] ; A Christopher,Boyd [5] ; June,Brand [5] ; Ruaridh,Buchan [6] ; Roberto,Calcedo [7] ; Paula,Carvelli [3] ; Mario,Chan [3] ; Seng H,Cheng [8] ; D David S,Collie [9] ; Steve,Cunningham [2] ; Heather E,Davidson [5] ; Gwyneth,Davies [3] ; Jane C,Davies [3] ; Lee A,Davies [10] ; Maria H,Dewar [6] ; Ann,Doherty [5] ; Jackie,Donovan [4] ; Natalie S,Dwyer [3] ; Hala I,Elgmati [6] ; Rosanna F,Featherstone [3] ; Jemyr,Gavino [3] ; Sabrina,Gea-Sorli [3] ; Duncan M,Geddes [4] ; James S R,Gibson [5] ; Deborah R,Gill [10] ; Andrew P,Greening [6] ; Uta,Griesenbach [3] ; David M,Hansell [4] ; Katharine,Harman [3] ; Tracy E,Higgins [3] ; Samantha L,Hodges [3] ; Stephen C,Hyde [10] ; Laura,Hyndman [5] ; J Alastair,Innes [6] ; Joseph,Jacob [4] ; Nancy,Jones [4] ; Brian F,Keogh [4] ; Maria P,Limberis [7] ; Paul,Lloyd-Evans [11] ; Alan W,Maclean [5] ; Michelle C,Manvell [3] ; Dominique,McCormick [10] ; Michael,McGovern [6] ; Gerry,McLachlan [9] ; Cuixiang,Meng [3] ; M Angeles,Montero [4] ; Hazel,Milligan [6] ; Laura J,Moyce [10] ; Gordon D,Murray [12] ; Andrew G,Nicholson [4] ; Tina,Osadolor [4] ; Javier,Parra-Leiton [5] ; David J,Porteous [5] ; Ian A,Pringle [10] ; Emma K,Punch [3] ; Kamila M,Pytel [3] ; Alexandra L,Quittner [13] ; Gina,Rivellini [3] ; Clare J,Saunders [3] ; Ronald K,Scheule [8] ; Sarah,Sheard [4] ; Nicholas J,Simmonds [4] ; Keith,Smith [11] ; Stephen N,Smith [3] ; Najwa,Soussi [3] ; Samia,Soussi [3] ; Emma J,Spearing [3] ; Barbara J,Stevenson [5] ; Stephanie G,Sumner-Jones [10] ; Minna,Turkkila [3] ; Rosa P,Ureta [3] ; Michael D,Waller [3] ; Marguerite Y,Wasowicz [3] ; James M,Wilson [7] ; Paul,Wolstenholme-Hogg [8] ; UK Cystic Fibrosis Gene Therapy Consortium
作者单位: Imperial College London, London, UK. Electronic address: e.alton@imperial.ac.uk. [1] Royal Hospital for Sick Children, Edinburgh, UK. [2] Imperial College London, London, UK. [3] Royal Brompton and Harefield NHS Foundation Trust, London, UK. [4] Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK. [5] Western General Hospital, Edinburgh, UK. [6] Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA. [7] Genzyme, a Sanofi Company, Framingham, MA, USA. [8] The Roslin Institute and R(D)SVS, University of Edinburgh, Edinburgh, UK. [9] Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK. [10] NHS Blood and Transplant, Bristol, UK. [11] Usher Institute of Population Health Sciences and Informatics and Centre for Population Health Sciences, University of Edinburgh, Edinburgh, UK. [12] University of Miami, Miami, FL, USA. [13]
DOI 10.1016/S2213-2600(15)00245-3
PMID 26149841
发布时间 2025-05-29
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The Lancet. Respiratory medicine

The Lancet. Respiratory medicine

2015年3卷9期

684-691页

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