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

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...

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Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4673100/
https://www.ncbi.nlm.nih.gov/pubmed/26149841
http://dx.doi.org/10.1016/S2213-2600(15)00245-3

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