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

T2 Lentivirus GM-CSF gene therapy ameliorates autoimmune pulmonary alveolar proteinosis

Autor: Alicia B. Byrne, R C Ashworth, A Bhargava, Eric W.F.W. Alton, N Atsumi, Charles A. Morgan, Deborah R. Gill, H Lund-Palau, Stephen C. Hyde, Ian A. Pringle, A Pilou, Uta Griesenbach, Mario Chan, Cuixiang Meng

Publikováno v: BTS/BALR/BLF Early Career Investigators awards symposium.

Autoimmune pulmonary alveolar proteinosis (aPAP) is a lung disease characterised by the progressive accumulation of surfactant, leading to respiratory failure, due to anti-granulocyte-macrophage colony-stimulating factor (GM-CSF) auto-antibodies. The

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::9bba3aacc54dc24d400fbcfa8ff43ffa
https://doi.org/10.1136/thorax-2018-212555.2

Ex Vivo and In Vivo Lentivirus-Mediated Transduction of Airway Epithelial Progenitor Cells

Autor: Steven L. Brody, Raymond Farley, Marguerite Y Wasowicz, Makoto Inoue, Eric W.F.W. Alton, Mamoru Hasegawa, Cuixiang Meng, Uta Griesenbach, Giulia Leoni, Mario Chan

Publikováno v: Current Gene Therapy. 15:581-590

A key challenge in pulmonary gene therapy for cystic fibrosis is to provide long-term correction of the genetic defect. This may be achievable by targeting airway epithelial stem/progenitor cells with an integrating vector. Here, we evaluated the abi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::7bb3c92bf48e8a66a11141a8ab7be514
https://doi.org/10.2174/1566523215666151016123625

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

171. Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus

Autor: Deborah R. Gill, Amit C. Nathwani, Stephen C. Hyde, Uta Griesenbach, Kamila M Pytel, Cuixiang Meng, Jenny McIntosh, Lee A. Davies, Ian A. Pringle, Mario Chan, Michael C. Paul-Smith, Mamoru Hasegawa, Eric W.F.W. Alton, Makoto Inoue

Publikováno v: Molecular Therapy. 23

We have previously shown that the lung can be used as “factory” for production of secreted proteins, which are released into the circulation, using Sendai virus-mediated gene transfer. However, gene expression was transient and repeated administr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::04880cd1960245d6a7c85343f70b17ec