Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease

por Taylor, Kristin M., Meyers, Elizabeth, Phipps, Michael, Kishnani, Priya S., Cheng, Seng H., Scheule, Ronald K., Moreland, Rodney J.
Publicado 2013

Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D

por Hartshorn, Kevan L, Webby, Richard, White, Mitchell R, Tecle, Tesfaldet, Pan, Clark, Boucher, Susan, Moreland, Rodney J, Crouch, Erika C, Scheule, Ronald K
Publicado 2008

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

por Clayton, Nicholas P, Nelson, Carol A, Weeden, Timothy, Taylor, Kristin M, Moreland, Rodney J, Scheule, Ronald K, Phillips, Lucy, Leger, Andrew J, Cheng, Seng H, Wentworth, Bruce M
Publicado 2014

Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease

por Marshall, John, Ashe, Karen M., Bangari, Dinesh, McEachern, KerryAnne, Chuang, Wei-Lien, Pacheco, Joshua, Copeland, Diane P., Desnick, Robert J., Shayman, James A., Scheule, Ronald K., Cheng, Seng H.
Publicado 2010

Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease

por Cabrera-Salazar, Mario A., DeRiso, Matthew, Bercury, Scott D., Li, Lingyun, Lydon, John T., Weber, William, Pande, Nilesh, Cromwell, Mandy A., Copeland, Diane, Leonard, John, Cheng, Seng H., Scheule, Ronald K.
Publicado 2012

Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease

por Ashe, Karen M., Bangari, Dinesh, Li, Lingyun, Cabrera-Salazar, Mario A., Bercury, Scott D., Nietupski, Jennifer B., Cooper, Christopher G. F., Aerts, Johannes M. F. G., Lee, Edward R., Copeland, Diane P., Cheng, Seng H., Scheule, Ronald K., Marshall, John
Publicado 2011

Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo

por Griesenbach, Uta, Kitson, Chris, Garcia, Sara Escudero, Farley, Raymond, Singh, Charanjit, Somerton, Luci, Painter, Hazel, Smith, Rbecca L, Gill, Deborah R, Hyde, Stephen C, Chow, Yu-Hua, Hu, Jim, Gray, Mike, Edbrooke, Mark, Ogilvie, Varrie, MacGregor, Gordon, Scheule, Ronald K, Cheng, Seng H, Caplen, Natasha J, Alton, Eric WFW
Publicado 2006

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

por Alton, Eric W F W, Armstrong, David K, Ashby, Deborah, Bayfield, Katie J, Bilton, Diana, Bloomfield, Emily V, Boyd, A Christopher, Brand, June, Buchan, Ruaridh, Calcedo, Roberto, Carvelli, Paula, Chan, Mario, Cheng, Seng H, Collie, D David S, Cunningham, Steve, Davidson, Heather E, Davies, Gwyneth, Davies, Jane C, Davies, Lee A, Dewar, Maria H, Doherty, Ann, Donovan, Jackie, Dwyer, Natalie S, Elgmati, Hala I, Featherstone, Rosanna F, Gavino, Jemyr, Gea-Sorli, Sabrina, Geddes, Duncan M, Gibson, James S R, Gill, Deborah R, Greening, Andrew P, Griesenbach, Uta, Hansell, David M, Harman, Katharine, Higgins, Tracy E, Hodges, Samantha L, Hyde, Stephen C, Hyndman, Laura, Innes, J Alastair, Jacob, Joseph, Jones, Nancy, Keogh, Brian F, Limberis, Maria P, Lloyd-Evans, Paul, Maclean, Alan W, Manvell, Michelle C, McCormick, Dominique, McGovern, Michael, McLachlan, Gerry, Meng, Cuixiang, Montero, M Angeles, Milligan, Hazel, Moyce, Laura J, Murray, Gordon D, Nicholson, Andrew G, Osadolor, Tina, Parra-Leiton, Javier, Porteous, David J, Pringle, Ian A, Punch, Emma K, Pytel, Kamila M, Quittner, Alexandra L, Rivellini, Gina, Saunders, Clare J, Scheule, Ronald K, Sheard, Sarah, Simmonds, Nicholas J, Smith, Keith, Smith, Stephen N, Soussi, Najwa, Soussi, Samia, Spearing, Emma J, Stevenson, Barbara J, Sumner-Jones, Stephanie G, Turkkila, Minna, Ureta, Rosa P, Waller, Michael D, Wasowicz, Marguerite Y, Wilson, James M, Wolstenholme-Hogg, Paul
Publicado 2015