Stephen N. Smith
National Institutes of Health
21 Papers
578 Citations
Stephen N. Smith is an academic researcher from National Institutes of Health. The author has contributed to research in topics: Cystic fibrosis & Cystic fibrosis transmembrane conductance regulator. The author has an hindex of 16, co-authored 21 publications. Previous affiliations of Stephen N. Smith include University of Queensland & University of Edinburgh.
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Papers
Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.
Eric W.F.W. Alton,M Stern,Raymond Farley,Adam Jaffe,Sharon Chadwick,J. Phillips,Jane C. Davies,Stephen N. Smith,JE Browning,Michael G. Davies,Margaret E. Hodson,Stephen R. Durham,D. Li,Peter K. Jeffery,M. Scallan,RP Balfour,Simon J. Eastman,Seng H. Cheng,Alan E. Smith,D. P. Meeker,Duncan M. Geddes +20 more
TL;DR: The safety and efficacy of cationic-lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis was studied in a double-blind placebo-controlled trial.
459
Non–invasive liposome–mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice
Eric W.F.W. Alton,Peter G. Middleton,Natasha J. Caplen,Stephen N. Smith,D. M. Steel,Felix M. Munkonge,Peter K. Jeffery,Duncan M. Geddes,Stephen L. Hart,Robert Williamson +9 more
TL;DR: Overall, a range of correction was seen with restoration of about 50% of the deficit between wild type mice and untreated cf/cf controls, suggesting that this may offer a therapeutic alternative to adenoviral therapies.
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Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
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,David 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,T Higgins,Samantha L Hodges,Stephen C. Hyde,Laura Hyndman,J. Alastair Innes,Joseph Jacob,Nancy Aaron Jones,Brian F Keogh,Maria P. Limberis,Paul Lloyd-Evans,Alan W Maclean,Michelle C Manvell,Dominique McCormick,Michael McGovern,Gerry McLachlan,C 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 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 +79 more
TL;DR: In this article, a randomized, double-blind, placebo-controlled, phase 2b trial was conducted to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis, where patients were randomly assigned to receive 5 mL of either nebulised pGM169/GL67A gene-liposome complex or 0·9% saline (placebo) every 28 days for 1 year.
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Cystic fibrosis in the mouse by targeted insertional mutagenesis.
Julia R. Dorin,Paul Dickinson,Eric W.F.W. Alton,Stephen N. Smith,Duncan M. Geddes,Barbara J Stevenson,Wendy L. Kimber,Stewart Fleming,Alan Richard Clarke,Martin L. Hooper,L. Anderson,Rosa S. P. Beddington,David J. Porteous +12 more
TL;DR: In vivo electrophysiology demonstrates the predicted defect in chloride ion transport in these mice and can distinguish between each genotype, and provides a valid model system for the development and testing of therapies for cystic fibrosis patients.
318
•Journal Article
A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction
Julia R. Dorin,R Farley,Sheila Webb,Stephen N. Smith,E. Farini,S. J. Delaney,Brandon J. Wainwright,Eric W.F.W. Alton,David J. Porteous +8 more
TL;DR: It is demonstrated that 5% of the normal level of Cftr gene expression results in a disproportionately large correction of the chloride ion transport defect and essentially complete rescue of the intestinal disease.
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