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

An immunocytochemical assay to detect human CFTR expression following gene transfer

Autor: Cordelia Stearns, A. Christopher Boyd, Lisa Jones, Deborah R. Gill, Heather E Davidson, Angus C. Nairn, Emma Holder, Alex Horsley, Ann Doherty, Abigail R. Wilson, Stephen C. Hyde, Michelle C Manvell, Rebecca Coles, Gerry McLachlan, Jane C. Davies, David Collie, David J. Porteous, Uta Griesenbach, Ian A. Pringle, Eric W.F.W. Alton, Robert D. Gray, Stephanie G. Sumner-Jones, Marguerite Y Wasowicz, James Sr Gibson

Publikováno v: Molecular and cellular probes. 23(6)

BACKGROUND: To assess gene therapy treatment for cystic fibrosis (CF) in clinical trials it is essential to develop robust assays that can accurately detect transgene expression in human airway epithelial cells. Our aim was to develop a reproducible

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f371bedd61a353fa9470c2261aa81caa
https://pubmed.ncbi.nlm.nih.gov/19615439