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

Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung

Autor: David Collie, Uta Griesenbach, Laura Hyndman, A. C. Boyd, Heather E Davidson, David J. Porteous, Stephanie G. Sumner-Jones, Ronald K. Scheule, Gerry McLachlan, Abigail R. Wilson, S C Hyde, Catherine Gordon, Darren J. Shaw, Christina Vrettou, J A Innes, R. Blundell, Ian A. Pringle, Emma Holder, Ann Doherty, Jane C. Davies, Alison Baker, Eric W.F.W. Alton, Lee A. Davies, Deborah R. Gill, S H Cheng, Rebecca Coles, Peter Tennant, Barbara J Stevenson, Hazel Painter

Publikováno v: Gene therapy. 18(10)

We use both large and small animal models in our pre-clinical evaluation of gene transfer agents (GTAs) for cystic fibrosis (CF) gene therapy. Here, we report the use of a large animal model to assess three non-viral GTAs: 25 kDa-branched polyethylen

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::430eaa691aebb1e78eab6ab7221960e5
http://ora.ox.ac.uk/objects/uuid:5e5123f5-13ed-440b-944d-3ceb5d07482e

Role of biophysical parameters on ex vivo and in vivo gene transfer to the airway epithelium by polyethylenimine/albumin complexes

Autor: Massimo Conese, Laura Hyndman, E. Copreni, Maurizio Di Cicco, Carsten Rudolph, Sante Di Gioia, A. Christopher Boyd, Joanna Rejman, Carla Colombo, Ida De Fino, Alessandra Bragonzi, Salvatore Carrabino, Ann Doherty

Publikováno v: Biomacromolecules. 9(3)

Efficient gene transfer to the airways by nonviral vectors is a function of different parameters, among which the size and the charge of the transfecting particles. The aim of this study was to determine the transfection efficiency of polyethylenimin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::75dbd21c35a2af6249b81bfb3a126784
https://pubmed.ncbi.nlm.nih.gov/18275149

HIV-1 Tat protein transduction domain peptide facilitates gene transfer in combination with cationic liposomes

Autor: Laura Hyndman, Jerome L. Lemoine, David J. Porteous, Leaf Huang, A. Christopher Boyd, Xinsheng Nan

Publikováno v: Journal of controlled release : official journal of the Controlled Release Society. 99(3)

The protein transduction domain (PTD) of the HIV-1 Tat protein can facilitate the cellular and nuclear uptake of macromolecular particles. Here, we demonstrate that incorporation without covalent linkage of a 17-amino acid PTD peptide into gene deliv

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::58be551078f327c2c5149f5ef73e7c6a
https://pubmed.ncbi.nlm.nih.gov/15451601

Role of Biophysical Parameters on ex Vivo and in Vivo Gene Transfer to the Airway Epithelium by Polyethylenimine/Albumin Complexes.

Autor: Sante Di Gioia, Joanna Rejman, Salvatore Carrabino, Ida De Fino, Carsten Rudolph, Ann Doherty, Laura Hyndman, Maurizio Di Cicco, Elena Copreni, Alessandra Bragonzi, Carla Colombo, A. Christopher Boyd, Massimo Conese

Publikováno v: Biomacromolecules; Feb2008, Vol. 9 Issue 3, p859-866, 8p