A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Autor: Eric WFW 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, 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 SR 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, on behalf of the UK Cystic Fibrosis Gene Therapy Consortium

Publikováno v: Efficacy and Mechanism Evaluation, Vol 3, Iss 5 (2016)

Background: Cystic fibrosis (CF) is a chronic, life-limiting disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene leading to abnormal airway surface ion transport, chronic lung infections, inflammation and eventual re

Externí odkaz: https://doaj.org/article/09b29fe1b155492fba24a177fe4be24c

Sputum Trace Metals Are Biomarkers of Inflammatory and Suppurative Lung Disease

Autor: Andrew P. Greening, Donald Noble, M. Imrie, Dennis St. J. O'Reilly, Robert D. Gray, A. Christopher Boyd, David J. Porteous, Andrew Duncan, J. Alastair Innes

Publikováno v: Gray, R D, Duncan, A, Noble, D, Imrie, M, O'Reilly, D S, Innes, J A, Porteous, D J, Greening, A P & Boyd, A C 2010, ' Sputum trace metals are biomarkers of inflammatory and suppurative lung disease ', Chest, vol. 137, no. 3, pp. 635-641 . https://doi.org/10.1378/chest.09-1047

Background: Induced sputum cytology and protein biomarkers can be used to assess airways infl ammation. Increases in sputum iron have been described in infl ammatory lung disease. We hypothesized that other sputum metals may be affected by airways in

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::51f73e8d586ccb5edf44db66efd50baf
https://doi.org/10.1378/chest.09-1047

Biomarkers for cystic fibrosis lung disease: Application of SELDI-TOF mass spectrometry to BAL fluid

Autor: Robert D. Gray, Andrew P. Greening, M. Imrie, David J. Porteous, Jane C. Davies, Thomas N. Hilliard, Gordon MacGregor, Eric W.F.W. Alton, J. Alastair Innes, Andrew Bush, A. Christopher Boyd

Publikováno v: Macgregor, G, Gray, R D, Hilliard, T N, Imrie, M, Boyd, A C, Alton, E W, Bush, A, Davies, J C, Innes, J A, Porteous, D J & Greening, A P 2008, ' Biomarkers for cystic fibrosis lung disease : Application of SELDI-TOF mass spectrometry to BAL fluid ', Journal of Cystic Fibrosis, vol. 7, no. 5, pp. 352-358 . https://doi.org/10.1016/j.jcf.2007.12.005

BackgroundFor cystic fibrosis (CF) patients there is a lack of good assays of disease activity and response to new therapeutic interventions, including gene therapy. Current measures of airways inflammation severity are insensitive or non-specific.Me

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::82344ebdcdbd2d66909d557113e8a8c2

Lung clearance index is a sensitive, repeatable and practical measure of airways disease in adults with cystic fibrosis

Autor: Jane C. Davies, Alex Horsley, Steve Cunningham, David J. Porteous, Kenneth A Macleod, J A Innes, Per M. Gustafsson, Andrew P. Greening, Eric W.F.W. Alton, Clare Saunders

Publikováno v: Thorax. 63:135-140

Lung clearance index (LCI) is a sensitive marker of early lung disease in children but has not been assessed in adults. Measurement is hindered by the complexity of the equipment required. The aims of this study were to assess performance of a novel

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::45e8b4490bf70ad76060cb3a2ac509d0
https://doi.org/10.1136/thx.2007.082628

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