Zobrazeno 1 - 10
of 121
pro vyhledávání: '"Ronald K. Scheule"'
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
Autor:
Nicholas P Clayton, Carol A Nelson, Timothy Weeden, Kristin M Taylor, Rodney J Moreland, Ronald K Scheule, Lucy Phillips, Andrew J Leger, Seng H Cheng, Bruce M Wentworth
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 3, Iss C (2014)
Pompe disease is an autosomal recessive disorder caused by a deficiency of acid α-glucosidase (GAA; EC 3.2.1.20) and the resultant progressive lysosomal accumulation of glycogen in skeletal and cardiac muscles. Enzyme replacement therapy using recom
Externí odkaz:
https://doaj.org/article/cc5392267a07419090c8a0e91d1a8ae4
Autor:
Kristin M Taylor, Elizabeth Meyers, Michael Phipps, Priya S Kishnani, Seng H Cheng, Ronald K Scheule, Rodney J Moreland
Publikováno v:
PLoS ONE, Vol 8, Iss 2, p e56181 (2013)
Pompe disease, also known as glycogen storage disease (GSD) type II, is caused by deficiency of lysosomal acid α-glucosidase (GAA). The resulting glycogen accumulation causes a spectrum of disease severity ranging from a rapidly progressive course t
Externí odkaz:
https://doaj.org/article/eb859b5cc58943a8b5ff18a9d84331be
Autor:
Seng H. Cheng, Ronald K. Scheule
Liposomal delivery systems for nucleic acids fall within the larger class of non-viral delivery vehicles. Chronologically, liposomal systems designed to encapsulate pDNA were among the first non-viral systems to demonstrate gene delivery. Plasmid DNA
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::df97725e1df0fcd713e90d1d0394c7ce
https://doi.org/10.1201/9781003076919-6
https://doi.org/10.1201/9781003076919-6
Autor:
J. S. Kaczmarek, M. Maderia, Ronald K. Scheule, Ingrid Mechin, S. Bercury, G. D. Hurlbut, R. Ziegler, A. Nair, J. Bajko, A. Majewski, K. S. Simon, Joseph D. Batchelor, B. J. Hilbert, P. Manavalan, K. Nagarajan, C. Duffy, S. Altmann, M. Kothe, J. Foley
Cystic fibrosis (CF) results from mutations within the gene encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), a transmembrane chloride channel found on the apical surface of epithelial cells. The most common CF-causing mutation
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::429cdfcd0325476fa4bb776f012e3d72
Autor:
Mario A Cabrera-Salazar, Matthew Deriso, Scott D Bercury, Lingyun Li, John T Lydon, William Weber, Nilesh Pande, Mandy A Cromwell, Diane Copeland, John Leonard, Seng H Cheng, Ronald K Scheule
Publikováno v:
PLoS ONE, Vol 7, Iss 8, p e43310 (2012)
Neuropathic Gaucher disease (nGD), also known as type 2 or type 3 Gaucher disease, is caused by a deficiency of the enzyme glucocerebrosidase (GC). This deficiency impairs the degradation of glucosylceramide (GluCer) and glucosylsphingosine (GluSph),
Externí odkaz:
https://doaj.org/article/ec8f627f58cc40f6871e8639ff2b14b9
Autor:
Karen M Ashe, Dinesh Bangari, Lingyun Li, Mario A Cabrera-Salazar, Scott D Bercury, Jennifer B Nietupski, Christopher G F Cooper, Johannes M F G Aerts, Edward R Lee, Diane P Copeland, Seng H Cheng, Ronald K Scheule, John Marshall
Publikováno v:
PLoS ONE, Vol 6, Iss 6, p e21758 (2011)
The neuropathic glycosphingolipidoses are a subgroup of lysosomal storage disorders for which there are no effective therapies. A potential approach is substrate reduction therapy using inhibitors of glucosylceramide synthase (GCS) to decrease the sy
Externí odkaz:
https://doaj.org/article/cd3e8f6aa28047d9b5d2eaa2ee1c6e5d
Autor:
John Marshall, Karen M Ashe, Dinesh Bangari, KerryAnne McEachern, Wei-Lien Chuang, Joshua Pacheco, Diane P Copeland, Robert J Desnick, James A Shayman, Ronald K Scheule, Seng H Cheng
Publikováno v:
PLoS ONE, Vol 5, Iss 11, p e15033 (2010)
Fabry disease is an X-linked glycosphingolipid storage disorder caused by a deficiency in the activity of the lysosomal hydrolase α-galactosidase A (α-gal). This deficiency results in accumulation of the glycosphingolipid globotriaosylceramide (GL-
Externí odkaz:
https://doaj.org/article/055bbfe4fb664b8e9074473425ff3afe
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
https://doi.org/10.1038/gt.2013.61
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
https://doi.org/10.1016/j.biomaterials.2013.09.023