rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice

rAAVrh74.MCK.GALGT2 is a surrogate gene therapy that inhibits muscular dystrophy in multiple animal models. Here, we report on a dose-response study of functional muscle GALGT2 expression as well as toxicity and biodistribution studies after systemic intravenous (i.v.) delivery of rAAVrh74.MCK.GALGT...

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Publicat a:Mol Ther Methods Clin Dev
Autors principals: Zygmunt, Deborah A., Xu, Rui, Jia, Ying, Ashbrook, Anna, Menke, Chelsea, Shao, Guohong, Yoon, Jung Hae, Hamilton, Sonia, Pisharath, Harshan, Bolon, Brad, Martin, Paul T.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2019
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6923506/
https://ncbi.nlm.nih.gov/pubmed/31890730
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.005
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