Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength

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...

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Veröffentlicht in:	PloS one 2021-03, Vol.16 (3), p.e0248721-e0248721
Hauptverfasser:	Martin, Paul T, Zygmunt, Deborah A, Ashbrook, Anna, Hamilton, Sonia, Packer, Davin, Birch, Sharla M, Bettis, Amanda K, Balog-Alvarez, Cynthia J, Guo, Lee-Jae, Nghiem, Peter P, Kornegay, Joe N
Format:	Artikel
Sprache:	eng
Schlagworte:	Animal models Biology and Life Sciences Biopsy Cardiac function Cardiac muscle Care and treatment Children Clinical trials Diseases Dogs Dystrophin Dystrophy Editing Females Gene therapy Genes Genetic variability Genotype & phenotype Glycosylation Golden retrievers Health aspects Histopathology Hospitals Kinases Laboratory animals Males Medicine Medicine and Health Sciences Methodology Molecular modelling Muscle strength Muscular dystrophy Musculoskeletal system Mutation Nervous system Neuromuscular junctions Neurosciences Pediatrics Phenotypes Physiological aspects Point mutation Proteins Research and Analysis Methods Reviews Skeletal muscle Utrophin Veterinary colleges Veterinary medicine Youth
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