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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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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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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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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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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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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Comprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in mice
Tousignant, J D, Gates, A L, Ingram, L A, Johnson, C L, Nietupski, J B, Cheng, S H, Eastman, S J, Scheule, R K
Published in Human gene therapy (10.12.2000)
Published in Human gene therapy (10.12.2000)
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Rapid identification of novel functional promoters for gene therapy
Pringle, Ian A., Gill, Deborah R., Connolly, Mary M., Lawton, Anna E., Hewitt, Anne-Marie, Nunez-Alonso, Graciela, Cheng, Seng H., Scheule, Ronald K., Davies, Lee A., Hyde, Stephen C.
Published in Journal of molecular medicine (Berlin, Germany) (01.12.2012)
Published in Journal of molecular medicine (Berlin, Germany) (01.12.2012)
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Contribution of Toll-like Receptor 9 Signaling to the Acute Inflammatory Response to Nonviral Vectors
Zhao, Hongmei, Hemmi, Hiraoki, Akira, Shizuo, Cheng, Seng H, Scheule, Ronald K, Yew, Nelson S
Published in Molecular therapy (01.02.2004)
Published in Molecular therapy (01.02.2004)
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Journal Article
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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Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
Griesenbach, Uta, Kitson, Chris, Escudero Garcia, Sara, 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 W F W
Published in Respiratory research (15.02.2006)
Published in Respiratory research (15.02.2006)
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Journal Article
The role of doxorubicin in non-viral gene transfer in the lung
Griesenbach, Uta, Meng, Cuixiang, Farley, Raymond, Gardner, Aaron, Brake, Maresa A, Frankel, Gad M, Gruenert, Dieter C, Cheng, Seng H, Scheule, Ronald K, Alton, Eric W.F.W
Published in Biomaterials (01.04.2009)
Published in Biomaterials (01.04.2009)
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