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Published in PloS one (20.08.2014)
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IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice
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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
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Published in Molecular genetics and metabolism (01.04.2012)
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Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice
Zhu, Yunxiang, Li, Xuemei, McVie-Wylie, Alison, Jiang, Canwen, Thurberg, Beth L, Raben, Nina, Mattaliano, Robert J, Cheng, Seng H
Published in Biochemical journal (01.08.2005)
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Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease
Hadaczek, Piotr, Stanek, Lisa, Ciesielska, Agnieszka, Sudhakar, Vivek, Samaranch, Lluis, Pivirotto, Philip, Bringas, John, O'Riordan, Catherine, Mastis, Bryan, San Sebastian, Waldy, Forsayeth, John, Cheng, Seng H, Bankiewicz, Krystof S, Shihabuddin, Lamya S
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Inhibition of osteoclastogenesis by prolyl hydroxylase inhibitor dimethyloxallyl glycine
Leger, Andrew J, Altobelli, Allison, Mosquea, Leocadia M, Belanger, Adam J, Song, Antonius, Cheng, Seng H, Jiang, Canwen, Yew, Nelson S
Published in Journal of bone and mineral metabolism (01.09.2010)
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Dimorphic effects of transforming growth factor-β signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome
Cook, Jason R, Clayton, Nicholas P, Carta, Luca, Galatioto, Josephine, Chiu, Emily, Smaldone, Silvia, Nelson, Carol A, Cheng, Seng H, Wentworth, Bruce M, Ramirez, Francesco
Published in Arteriosclerosis, thrombosis, and vascular biology (01.04.2015)
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A bispecific protein capable of engaging CTLA-4 and MHCII protects non-obese diabetic mice from autoimmune diabetes
Zhao, Hongmei, Karman, Jozsef, Jiang, Ji-Lei, Zhang, Jinhua, Gumlaw, Nathan, Lydon, John, Zhou, Qun, Qiu, Huawei, Jiang, Canwen, Cheng, Seng H, Zhu, Yunxiang
Published in PloS one (21.05.2013)
Published in PloS one (21.05.2013)
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Increased Hepatic Insulin Action in Diet-Induced Obese Mice Following Inhibition of Glucosylceramide Synthase
Yew, Nelson S, Zhao, Hongmei, Hong, Eun-Gyoung, Wu, I-Huan, Przybylska, Malgorzata, Siegel, Craig, Shayman, James A, Arbeeny, Cynthia M, Kim, Jason K, Jiang, Canwen, Cheng, Seng H
Published in PloS one (21.06.2010)
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Reducing glycosphingolipid biosynthesis in airway cells partially ameliorates disease manifestations in a mouse model of asthma
Karman, Jozsef, Tedstone, Jennifer L., Gumlaw, Nathan K., Zhu, Yunxiang, Yew, Nelson, Siegel, Craig, Guo, Shuling, Siwkowski, Andrew, Ruzek, Melanie, Jiang, Canwen, Cheng, Seng H.
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Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
Schulz, Martin, Levy, Daniel I., Petropoulos, Christos J., Bashirians, George, Winburn, Ian, Mahn, Matthias, Somanathan, Suryanarayan, Cheng, Seng H., Byrne, Barry J.
Published in Molecular therapy (01.03.2023)
Published in Molecular therapy (01.03.2023)
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Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse
Passini, Marco A., Bu, Jie, Fidler, Jonathan A., Ziegler, Robin J., Foley, Joseph W., Dodge, James C., Yang, Wendy W., Clarke, Jennifer, Taksir, Tatyana V., Griffiths, Denise A., Zhao, Michael A., O'Riordan, Catherine R., Schuchman, Edward H., Shihabuddin, Lamya S., Cheng, Seng H.
Published in Proceedings of the National Academy of Sciences - PNAS (29.05.2007)
Published in Proceedings of the National Academy of Sciences - PNAS (29.05.2007)
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Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease
James C. Dodge, Jennifer Clarke, Antonius Song, Jie Bu, Yang, Wendy, Tatyana V. Taksir, Denise Griffiths, Michael A. Zhao, Schuchman, Edward H., Cheng, Seng H., Catherine R. O'Riordan, Shihabuddin, Lamya S., Marco A. Passini, Stewart, Gregory R.
Published in Proceedings of the National Academy of Sciences - PNAS (06.12.2005)
Published in Proceedings of the National Academy of Sciences - PNAS (06.12.2005)
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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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The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo
Kyostio-Moore, Sirkka, Berthelette, Patricia, Piraino, Susan, Sookdeo, Cathleen, Nambiar, Bindu, Jackson, Robert, Burnham, Brenda, O'Riordan, Catherine R, Cheng, Seng H, Armentano, Donna
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Stabilization of Vascular Endothelial Growth Factor mRNA by Hypoxia-Inducible Factor 1
Liu, Louis X., Lu, Hsienwie, Luo, Yuxia, Date, Taro, Belanger, Adam J., Vincent, Karen A., Akita, Geoffrey Y., Goldberg, Mark, Cheng, Seng H., Gregory, Richard J., Jiang, Canwen
Published in Biochemical and biophysical research communications (08.03.2002)
Published in Biochemical and biophysical research communications (08.03.2002)
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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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Reduced Inflammatory Response to Plasmid DNA Vectors by Elimination and Inhibition of Immunostimulatory CpG Motifs
Yew, Nelson S., Zhao, Hongmei, Wu, I-Huan, Song, Antonius, Tousignant, Jennifer D., Przybylska, Malgorzata, Cheng, Seng H.
Published in Molecular therapy (01.03.2000)
Published in Molecular therapy (01.03.2000)
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