Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MCK.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength

Paul Martin, Deborah Zygmunt, Anna Ashbrook, Sonia Hamilton, Davin Packer, Sharla Birch, Amanda Bettis, Cynthia Balog-Alvarez, Lee-Jae Guo, Peter Nghiem & Joe Kornegay
We have examined the effects of intravenous (IV) delivery of rAAVrh74.MHCK7.GALGT2 in the golden retriever muscular dystrophy (GRMD) model of Duchenne Muscular Dystrophy (DMD). After baseline testing, GRMD dogs were treated at 3 months of age and reassessed at 6 months. This 3-6 month age range is a period of rapid disease progression, thus offering a relatively short window to establish treatment efficacy. Measures analyzed included muscle AAV transduction, GALGT2 transgene expression, GALGT2-induced glycosylation, muscle...
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