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Non-invasive Intravenous Administration of AAV9 Transducing Iduronate Sulfatase Leads to Global Metabolic Correction and Prevention of Neurologic Deficits in a Mouse Model of Hunter Syndrome

Kanut Laoharawee,Kelly M. Podetz-Pedersen,Tam T. Nguyen,Sajya M. Singh,Miles C. Smith,Lalitha R. Belur, Walter C. Low,Karen F. Kozarsky,R. Scott McIvor

MOLECULAR GENETICS AND METABOLISM REPORTS（2023）

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Key words
Hunter syndrome,Metabolic disorder,Mucopolysaccharidosis type II,Gene therapy,Gene transfer,MPS II
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