Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency by Patricia Lam, Anna Ashbrook, Deborah A. Zygmunt, Cong Yan, Hong Du, Paul T. Martin

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rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice by Deborah A. Zygmunt, Rui Xu, Ying Jia, Anna Ashbrook, Chelsea Menke, Guohong Shao, Jung Hae Yoon, Sonia Hamilton, Harshan Pisharath, Brad Bolon, Paul T. Martin

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Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effec... by Paul T Martin, Deborah A Zygmunt, Anna Ashbrook, Sonia Hamilton, Davin Packer, Sharla M Birch, Amanda K Bettis, Cynthia J Balog-Alvarez, Lee-Jae Guo, Peter P Nghiem, Joe N Kornegay

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A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2 by Kevin M. Flanigan, Tatyana A. Vetter, Tabatha R. Simmons, Megan Iammarino, Emma C. Frair, Federica Rinaldi, Louis G. Chicoine, Johan Harris, John P. Cheatham, Sharon L. Cheatham, Brian Boe, Megan A. Waldrop, Deborah A. Zygmunt, Davin Packer, Paul T. Martin

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An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque by Rui Xu, Ying Jia, Deborah A. Zygmunt, Megan L. Cramer, Kelly E. Crowe, Guohong Shao, Agatha E. Maki, Haley N. Guggenheim, Benjamin C. Hood, Danielle A. Griffin, Ellyn Peterson, Brad Bolon, John P. Cheatham, Sharon L. Cheatham, Kevin M. Flanigan, Louise R. Rodino-Klapac, Louis G. Chicoine, Paul T. Martin

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