Pharmacological Characterization of a Novel ENaCα siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis

Autor: Kenneth L Clark, Stephen A Hughes, Pallav Bulsara, Jill Coates, Kitty Moores, Joel Parry, Michael Carr, Ruth J Mayer, Paul Wilson, Chris Gruenloh, Daren Levin, Jill Darton, Wolf-Michael Weber, Katja Sobczak, Deborah R Gill, Stephen C Hyde, Lee A Davies, Ian A Pringle, Stephanie G Sumner-Jones, Vasant Jadhav, Sharon Jamison, Walter R Strapps, Victoria Pickering, Mark R Edbrooke

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 2, Iss C (2013)

Lung pathology in cystic fibrosis is linked to dehydration of the airways epithelial surface which in part results from inappropriately raised sodium reabsorption through the epithelial sodium channel (ENaC). To identify a small-interfering RNA (siRN

Externí odkaz: https://doaj.org/article/b78fb7e93ba44314a82b90606a64ce1b

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung

Autor: A. C. Boyd, Eric W.F.W. Alton, Stephanie G. Sumner-Jones, T Higgins, S C Hyde, Deborah R. Gill, Lee A. Davies, A Dayan, Gerry McLachlan, David J. Porteous, S H Cheng, Jane C. Davies, Uta Griesenbach, Ronald K. Scheule, J A Innes, Ian A. Pringle

Publikováno v: Gene Therapy. 21:89-95

For gene therapy to improve lung function in cystic fibrosis (CF) subjects, repeated administration of the gene transfer agent over the lifetime of patients is likely to be necessary. This requirement limits the utility of adenoviral and adeno-associ

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::72372e01f57e2fb7b68ce17f954700d6
https://doi.org/10.1038/gt.2013.61

The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep

Autor: J. Alastair Innes, Dominique McCormick, T Higgins, Heather E Davidson, Ronald K. Scheule, Deborah R. Gill, A. Christopher Boyd, Darren J. Shaw, Ian A. Pringle, Catherine Gordon, David J. Porteous, Eilidh Baker, Gerry McLachlan, Jane C. Davies, Rebecca Coles, S. C. Hyde, Eric W.F.W. Alton, Peter Tennant, David Collie, Uta Griesenbach, Alison Baker, Sionagh Smith, Michael McGovern, Stephanie G. Sumner-Jones, Seng H. Cheng, Christina Vrettou

Publikováno v: Alton, E W, Baker, A, Baker, E, Boyd, C, Cheng, S H, Coles, R, Collie, D, Davidson, H, Davies, J C, Gill, D R, Gordon, C, Griesenbach, U, Higgins, T, Hyde, S C, Innes, J A, McCormick, D, McGovern, M, McLachlan, G, Porteous, D, Pringle, I A, Scheule, R K, Shaw, D, Smith, S, Summer-Jones, S, Tennant, P & Vrettou, C 2013, ' The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep ', Biomaterials, vol. 34, no. 38, pp. 10267-10277 . https://doi.org/10.1016/j.biomaterials.2013.09.023

Clinically effective gene therapy for Cystic Fibrosis has been a goal for over 20 years. A plasmid vector (pGM169) that generates persistent expression and reduced host inflammatory responses in mice has raised prospects for translation to the clinic

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::193abd643570f53d00904ebe8e3517e4
https://doi.org/10.1016/j.biomaterials.2013.09.023

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

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

Autor: Stephen C. Hyde, David J. Porteous, Uta Griesenbach, Deborah R. Gill, Mario Chan, Stephanie G Sumner-Jones, Syahril Abdullah, Ian A. Pringle, Reto P. Bazzani, Lee A. Davies, Anne-Marie Green, Felix M. Munkonge, Anna E. Lawton, Anusha Varathalingam, Jane C. Davies, Graciela A Nunez-Alonso, Eric W.F.W. Alton, A. Christopher Boyd, Seng H. Cheng, Nelson S. Yew, Hongyu Li, David N. Sheppard

Publikováno v: Nature Biotechnology. 26:549-551

Pulmonary delivery of plasmid DNA (pDNA)/cationic liposome complexes is associated with an acute unmethylated CG dinucleotide (CpG)-mediated inflammatory response and brief duration of transgene expression. We demonstrate that retention of even a sin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::727751e896d0cf47ef864ebe680365ff
https://doi.org/10.1038/nbt1399