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

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

Publikováno v: Alton, E W F W, Armstrong, D K, Ashby, D, Bayfield, K J, Bilton, D, Bloomfield, E V, Boyd, A C, Brand, J, Buchan, R, Calcedo, R, Carvelli, P, Chan, M, Cheng, S H, Collie, D D S, Cunningham, S, Davidson, H E, Davies, G, Davies, J C, Davies, L A, Dewar, M H, Doherty, A, Donovan, J, Dwyer, N S, Elgmati, H I, Featherstone, R F, Gavino, J, Gea-sorli, S, Geddes, D M, Gibson, J S R, Gill, D R, Greening, A P, Griesenbach, U, Hansell, D M, Harman, K, Higgins, T E, Hodges, S L, Hyde, S C, Hyndman, L, Innes, J A, Jacob, J, Jones, N, Keogh, B F, Limberis, M P, Lloyd-evans, P, Maclean, A W, Manvell, M C, McCormick, D, McGovern, M, McLachlan, G, Meng, C, Montero, M A, Milligan, H, Moyce, L J, Murray, G D, Nicholson, A G, Osadolor, T, Parra-leiton, J, Porteous, D J, Pringle, I A, Punch, E K, Pytel, K M, Quittner, A L, Rivellini, G, Saunders, C J, Scheule, R K, Sheard, S, Simmonds, N J, Smith, K, Smith, S N, Soussi, N, Soussi, S, Spearing, E J, Stevenson, B J, Sumner-jones, S G, Turkkila, M, Ureta, R P, Waller, M D, Wasowicz, M Y, Wilson, J M & Wolstenholme-hogg, P 2015, ' 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, vol. 3, no. 9, pp. 684-691 . https://doi.org/10.1016/S2213-2600(15)00245-3
The Lancet. Respiratory Medicine

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 cyst

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::bf873b19e7113b67bec0cf60e5082675
https://hdl.handle.net/20.500.11820/3d999f62-1304-4834-9d9b-d00a015a955d