Article
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.
The Lancet. Respiratory medicine - 1 Sept 2015
Alton Eric W F W, Armstrong David K, Ashby Deborah, Bayfield Katie J, Bilton Diana, Bloomfield Emily V, Boyd A Christopher, Brand June, Buchan Ruaridh, Calcedo Roberto, Carvelli Paula, Chan Mario, Cheng Seng H, Collie D David S, Cunningham Steve, Davidson Heather E, Davies Gwyneth, Davies Jane C, Davies Lee A, Dewar Maria H, Doherty Ann, Donovan Jackie, Dwyer Natalie S, Elgmati Hala I, Featherstone Rosanna F, Gavino Jemyr, Gea-Sorli Sabrina, Geddes Duncan M, Gibson James S R, Gill Deborah R, Greening Andrew P, Griesenbach Uta, Hansell David M, Harman Katharine, Higgins Tracy E, Hodges Samantha L, Hyde Stephen C, Hyndman Laura, Innes J Alastair, Jacob Joseph, Jones Nancy, Keogh Brian F, Limberis Maria P, Lloyd-Evans Paul, Maclean Alan W, Manvell Michelle C, McCormick Dominique, McGovern Michael, McLachlan Gerry, Meng Cuixiang, Montero M Angeles, Milligan Hazel, Moyce Laura J, Murray Gordon D, Nicholson Andrew G, Osadolor Tina, Parra-Leiton Javier, Porteous David J, Pringle Ian A, Punch Emma K, Pytel Kamila M, Quittner Alexandra L, Rivellini Gina, Saunders Clare J, Scheule Ronald K, Sheard Sarah, Simmonds Nicholas J, Smith Keith, Smith Stephen N, Soussi Najwa, Soussi Samia, Spearing Emma J, Stevenson Barbara J, Sumner-Jones Stephanie G, Turkkila Minna, Ureta Rosa P, Waller Michael D, Wasowicz Marguerite Y, Wilson James M, Wolstenholme-Hogg Paul
Abstract excerpt
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, double-blind, placebo-controlled, phase 2b trial in two cystic fibrosis centres with patients recruited from 18 sites in...
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