Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease
作者： Jeong-A Lim , Haiqing Yi , Fengqin Gao , Nina Raben , Priya S. Kishnani
DOI: 10.1016/J.OMTM.2019.01.006
关键词: Skeletal muscle 、 Spinal cord 、 Glycogen storage disease type II 、 Virus 、 Enzyme replacement therapy 、 Endocrinology 、 Vector (molecular biology) 、 Glycogen 、 Internal medicine 、 Genetic enhancement 、 Medicine
摘要: … For systemic delivery of the GAA transgene, we chose the AAV-PHP.B vector that was … intravenously injected with the AAV-PHP.B-GAA vector at a dose of 5 × 10 12 vg/kg (GAAKO-AAV), …
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