Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease
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
Published in PloS one (17.08.2012)
Published in PloS one (17.08.2012)
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Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease
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
Published in PloS one (24.11.2010)
Published in PloS one (24.11.2010)
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Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease
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
Published in PloS one (29.06.2011)
Published in PloS one (29.06.2011)
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Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
Ashe, Karen M, Budman, Eva, Bangari, Dinesh S, Siegel, Craig S, Nietupski, Jennifer B, Wang, Bing, Desnick, Robert J, Scheule, Ronald K, Leonard, John P, Cheng, Seng H, Marshall, John
Published in Molecular medicine (Cambridge, Mass.) (30.04.2015)
Published in Molecular medicine (Cambridge, Mass.) (30.04.2015)
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Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
Hurlbut, Gregory D, Ziegler, Robin J, Nietupski, Jennifer B, Foley, Joseph W, Woodworth, Lisa A, Meyers, Elizabeth, Bercury, Scott D, Pande, Nilesh N, Souza, David W, Bree, Mark P, Lukason, Michael J, Marshall, John, Cheng, Seng H, Scheule, Ronald K
Published in Molecular therapy (01.11.2010)
Published in Molecular therapy (01.11.2010)
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Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D
Hartshorn, Kevan L, Webby, Richard, White, Mitchell R, Tecle, Tesfaldet, Pan, Clark, Boucher, Susan, Moreland, Rodney J, Crouch, Erika C, Scheule, Ronald K
Published in Respiratory research (23.09.2008)
Published in Respiratory research (23.09.2008)
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Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
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
Published in Molecular therapy. Nucleic acids (01.10.2014)
Published in Molecular therapy. Nucleic acids (01.10.2014)
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Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice
Nietupski, Jennifer B., Pacheco, Joshua J., Chuang, Wei-Lien, Maratea, Kimberly, Li, Lingyun, Foley, Joseph, Ashe, Karen M., Cooper, Christopher G.F., Aerts, Johannes M.F.G., Copeland, Diane P., Scheule, Ronald K., Cheng, Seng H., Marshall, John
Published in Molecular genetics and metabolism (01.04.2012)
Published in Molecular genetics and metabolism (01.04.2012)
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Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
Nietupski, Jennifer B, Hurlbut, Gregory D, Ziegler, Robin J, Chu, Qiuming, Hodges, Bradley L, Ashe, Karen M, Bree, Mark, Cheng, Seng H, Gregory, Richard J, Marshall, John, Scheule, Ronald K
Published in Molecular therapy (01.11.2011)
Published in Molecular therapy (01.11.2011)
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The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
Alton, Eric W.F.W, Baker, Alison, Baker, Eilidh, Boyd, A. Christopher, Cheng, Seng H, Coles, Rebecca L, Collie, D. David S, Davidson, Heather, Davies, Jane C, Gill, Deborah R, Gordon, Catherine, Griesenbach, Uta, Higgins, Tracy, Hyde, Stephen C, Innes, J. Alastair, McCormick, Dominique, McGovern, Michael, McLachlan, Gerry, Porteous, David J, Pringle, Ian, Scheule, Ronald K, Shaw, Darren J, Smith, Sionagh, Sumner-Jones, Stephanie G, Tennant, Peter, Vrettou, Christina
Published in Biomaterials (01.12.2013)
Published in Biomaterials (01.12.2013)
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Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion
Ziegler, Robin J., Salegio, Ernesto A., Dodge, James C., Bringas, John, Treleaven, Christopher M., Bercury, Scott D., Tamsett, Thomas J., Shihabuddin, Lamya, Hadaczek, Piotr, Fiandaca, Massimo, Bankiewicz, Krystof, Scheule, Ronald K.
Published in Experimental neurology (01.10.2011)
Published in Experimental neurology (01.10.2011)
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Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease
Cabrera-Salazar, Mario A, Roskelley, Eric M, Bu, Jie, Hodges, Bradley L, Yew, Nelson, Dodge, James C, Shihabuddin, Lamya S, Sohar, Istvan, Sleat, David E, Scheule, Ronald K, Davidson, Beverly L, Cheng, Seng H, Lobel, Peter, Passini, Marco A
Published in Molecular therapy (01.10.2007)
Published in Molecular therapy (01.10.2007)
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Intratracheal Recombinant Surfactant Protein D Prevents Endotoxin Shock in the Newborn Preterm Lamb
Ikegami, Machiko, Carter, Karen, Bishop, Kimberly, Yadav, Annuradha, Masterjohn, Elizabeth, Brondyk, William, Scheule, Ronald K, Whitsett, Jeffrey A
Published in American journal of respiratory and critical care medicine (15.06.2006)
Published in American journal of respiratory and critical care medicine (15.06.2006)
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