Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

Autor: Alton EWFW; Imperial College London, London, UK. Electronic address: e.alton@imperial.ac.uk., Armstrong DK; Royal Hospital for Sick Children, Edinburgh, UK., Ashby D; Imperial College London, London, UK., Bayfield KJ; Imperial College London, London, UK., Bilton D; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Bloomfield EV; Imperial College London, London, UK., Boyd AC; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Brand J; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Buchan R; Western General Hospital, Edinburgh, UK., Calcedo R; Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA., Carvelli P; Imperial College London, London, UK., Chan M; Imperial College London, London, UK., Cheng SH; Genzyme, a Sanofi Company, Framingham, MA, USA., Collie DDS; The Roslin Institute and R(D)SVS, University of Edinburgh, Edinburgh, UK., Cunningham S; Royal Hospital for Sick Children, Edinburgh, UK., Davidson HE; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Davies G; Imperial College London, London, UK., Davies JC; Imperial College London, London, UK., Davies LA; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Dewar MH; Western General Hospital, Edinburgh, UK., Doherty A; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Donovan J; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Dwyer NS; Imperial College London, London, UK., Elgmati HI; Western General Hospital, Edinburgh, UK., Featherstone RF; Imperial College London, London, UK., Gavino J; Imperial College London, London, UK., Gea-Sorli S; Imperial College London, London, UK., Geddes DM; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Gibson JSR; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Gill DR; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Greening AP; Western General Hospital, Edinburgh, UK., Griesenbach U; Imperial College London, London, UK., Hansell DM; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Harman K; Imperial College London, London, UK., Higgins TE; Imperial College London, London, UK., Hodges SL; Imperial College London, London, UK., Hyde SC; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Hyndman L; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Innes JA; Western General Hospital, Edinburgh, UK., Jacob J; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Jones N; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Keogh BF; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Limberis MP; Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA., Lloyd-Evans P; NHS Blood and Transplant, Bristol, UK., Maclean AW; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Manvell MC; Imperial College London, London, UK., McCormick D; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., McGovern M; Western General Hospital, Edinburgh, UK., McLachlan G; The Roslin Institute and R(D)SVS, University of Edinburgh, Edinburgh, UK., Meng C; Imperial College London, London, UK., Montero MA; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Milligan H; Western General Hospital, Edinburgh, UK., Moyce LJ; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Murray GD; Usher Institute of Population Health Sciences and Informatics and Centre for Population Health Sciences, University of Edinburgh, Edinburgh, UK., Nicholson AG; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Osadolor T; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Parra-Leiton J; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Porteous DJ; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Pringle IA; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Punch EK; Imperial College London, London, UK., Pytel KM; Imperial College London, London, UK., Quittner AL; University of Miami, Miami, FL, USA., Rivellini G; Imperial College London, London, UK., Saunders CJ; Imperial College London, London, UK., Scheule RK; Genzyme, a Sanofi Company, Framingham, MA, USA., Sheard S; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Simmonds NJ; Royal Brompton and Harefield NHS Foundation Trust, London, UK., Smith K; NHS Blood and Transplant, Bristol, UK., Smith SN; Imperial College London, London, UK., Soussi N; Imperial College London, London, UK., Soussi S; Imperial College London, London, UK., Spearing EJ; Imperial College London, London, UK., Stevenson BJ; Centre for Genomic and Experimental Medicine, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK., Sumner-Jones SG; Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK., Turkkila M; Imperial College London, London, UK., Ureta RP; Imperial College London, London, UK., Waller MD; Imperial College London, London, UK., Wasowicz MY; Imperial College London, London, UK., Wilson JM; Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wolstenholme-Hogg P; Genzyme, a Sanofi Company, Framingham, MA, USA.

Publikováno v: The Lancet. Respiratory medicine [Lancet Respir Med] 2015 Sep; Vol. 3 (9), pp. 684-691. Date of Electronic Publication: 2015 Jul 03.

A phase I/IIa safety and efficacy study of nebulized liposome-mediated gene therapy for cystic fibrosis supports a multidose trial

Autor: Alton, E. W. F. W., Boyd, A. C., Porteous, D. J., Davies, G., Davies, J. C., Griesenbach, U., Higgins, T. E., Gill, D. R., Hyde, S. C., Innes, J. A., Katie Bayfield, Bell, N., Brand, J., Brum, A., Burney, T., Calcedo, R., Carvelli, P., Chalmers, F., Chan, M., Cheng, S. H., Collie, D. D. S., Cunningham, S., Davidson, H., Davies, L. A., Davis, S., Dewar, M., Doherty, A., Donovan, J., Dwyer, N. S., Embley, M., Geddes, D. M., Gibson, J. S. R., Greening, A. P., Hansell, D. M., Hellings, L., Hyndman, L., Jacob, J., Jones, N., Keogh, B. F., Lees, B., Limberis, M. P., Lloyd-Evans, P., Manvell, M. C., Mccormick, D., Mclachlan, G., Mclean, F., Meng, C., Murray, G. D., Newman, N., Nicholson, A. G., Parra-Leiton, J., Pringle, I. A., Reid, P., Rivellini, G., Saunders, C. J., Scheule, R. K., Sheard, S., Sheridan, H., Simpson, A., Smith, K., Smith, S. N., Soussi, S., Stevenson, B., Sumner-Jones, S. G., Voase, N., Wasowicz, M. Y., Wilson, A., Wilson, J. M., Wolstenholme-Hogg, P., Yanez-Lopez, M.

Publikováno v: Scopus-Elsevier

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::33360848c217cc2a4ab6c9eaecfc1e61
http://www.scopus.com/inward/record.url?eid=2-s2.0-84949035059&partnerID=MN8TOARS

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Factors Associated with Worse Lung Function in Cystic Fibrosis Patients with Persistent Staphylococcus aureus.

Autor: Junge, Sibylle¹, Görlich, Dennis², den Reijer, Martijn³, Wiedemann, Bärbel⁴, Tümmler, Burkhard⁵, Ellemunter, Helmut⁶, Dübbers, Angelika⁷, Küster, Peter⁸, Ballmann, Manfred⁹, Koerner-Rettberg, Cordula⁹, Große-Onnebrink, Jörg¹⁰, Heuer, Eberhardt¹¹, Sextro, Wolfgang¹¹, Mainz, Jochen G.¹², Hammermann, Jutta¹³, Riethmüller, Joachim¹⁴, Graepler-Mainka, Ute¹⁴, Staab, Doris¹⁵, Wollschläger, Bettina¹⁶, Szczepanski, Rüdiger¹⁷

Publikováno v: PLoS ONE. 11/18/2016, Vol. 11 Issue 11, p1-18. 18p.

Lung clearance index in adults with non-cystic fibrosis bronchiectasis.

Autor: Gonem, Sherif^1,2 sg330@le.ac.uk, Scadding, Alys¹, Soares, Marcia¹, Singapuri, Amisha¹, Gustafsson, Per³, Ohri, Chandra¹, Range, Simon¹, Brightling, Christopher E.¹, Pavord, Ian¹, Horsley, Alex^4,5, Siddiqui, Salman¹

Publikováno v: Respiratory Research. 2014, Vol. 15 Issue 5, p1-9. 9p. 4 Charts, 4 Graphs.