Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.
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]
医学主题词
投药, 吸入(Administration, Inhalation);青少年(Adolescent);成年人(Adult);儿童(Child);囊性纤维化(Cystic Fibrosis);囊性纤维化跨膜传导调节因子(Cystic Fibrosis Transmembrane Conductance Regulator);双盲法(Double-Blind Method);女(雌)性(Female);用力呼气量(Forced Expiratory Volume);人类(Humans);脂质体(Liposomes);男(雄)性(Male);突变(Mutation);雾化器和汽化器(Nebulizers and Vaporizers);质粒(Plasmids);青年人(Young Adult)
DOI
10.1016/S2213-2600(15)00245-3
PMID
26149841
发布时间
2025-05-29
- 浏览9
The Lancet. Respiratory medicine
The Lancet. Respiratory medicine
2015年3卷9期
684-691页
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