Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease von 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

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Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease von 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

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Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease von 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

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Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease von Taylor, Kristin M, Meyers, Elizabeth, Phipps, Michael, Kishnani, Priya S, Cheng, Seng H, Scheule, Ronald K, Moreland, Rodney J

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Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease von 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

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Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy von 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

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Surfactant Protein-D Inhibits Lung Inflammation Caused by Ventilation in Premature Newborn Lambs von SATO, Atsuyasu, WHITSETT, Jeffrey A, SCHEULE, Ronald K, LKEGAMI, Machiko

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Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D von Hartshorn, Kevan L, Webby, Richard, White, Mitchell R, Tecle, Tesfaldet, Pan, Clark, Boucher, Susan, Moreland, Rodney J, Crouch, Erika C, Scheule, Ronald K

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

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Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice von 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

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Systemic Insulin-like Growth Factor-1 Reverses Hypoalgesia and Improves Mobility in a Mouse Model of Diabetic Peripheral Neuropathy von Chu, Qiuming, Moreland, Rod, Yew, Nelson S, Foley, Joseph, Ziegler, Robin, Scheule, Ronald K

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Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression von 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

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Surfactant Protein-D and Surfactant Inhibit Endotoxin-Induced Pulmonary Inflammation von Ikegami, Machiko, Scoville, Elizabeth A., Grant, Shawn, Korfhagen, Thomas, Brondyk, William, Scheule, Ronald K., Whitsett, Jeffrey A.

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Surfactant protein-D regulates the postnatal maturation of pulmonary surfactant lipid pool sizes von Ikegami, Machiko, Grant, Shawn, Korfhagen, Thomas, Scheule, Ronald K, Whitsett, Jeffrey A

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The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep von 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

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Long-term Transgene Expression from Plasmid DNA Gene Therapy Vectors Is Negatively Affected by CpG Dinucleotides von Hodges, Bradley L, Taylor, Kristin M, Joseph, Macy F, Bourgeois, Sarah A, Scheule, Ronald K

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The role of CpG motifs in immunostimulation and gene therapy von Scheule, Ronald K

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Comprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in mice von TOUSIGNANT, Jennifer D, GATES, Amy L, INGRAM, Laurie A, JOHNSON, Carrie L, NIETUPSKI, Jennifer B, CHENG, Seng H, EASTMAN, Simon J, SCHEULE, Ronald K

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Intratracheal Recombinant Surfactant Protein D Prevents Endotoxin Shock in the Newborn Preterm Lamb von Ikegami, Machiko, Carter, Karen, Bishop, Kimberly, Yadav, Annuradha, Masterjohn, Elizabeth, Brondyk, William, Scheule, Ronald K, Whitsett, Jeffrey A

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CpG-Depleted Plasmid DNA Vectors with Enhanced Safety and Long-Term Gene Expression in Vivo von Yew, Nelson S., Zhao, Hongmei, Przybylska, Malgorzata, Wu, I-Huan, Tousignant, Jennifer D., Scheule, Ronald K., Cheng, Seng H.

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