Intra-articular AAV9 α-iduronidase gene therapy in the canine model of mucopolysaccharidosis type I results in rapid synovial and cartilage iduronidase expression, clearance of heparan sulfate, and high serum α-iduronidase levels
Autor: | Haoyue Zhang, Raymond Y. Wang, Jackie K. Jens, Elizabeth M. Snella, Patricia I. Dickson, Sarah P. Young, Shih-hsin Kan, Afshin Aminian, N. Matthew Ellinwood |
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Rok vydání: | 2019 |
Předmět: |
Chemistry
Endocrinology Diabetes and Metabolism Cartilage Genetic enhancement High serum Heparan sulfate Biochemistry Molecular biology Mucopolysaccharidosis type I chemistry.chemical_compound Endocrinology medicine.anatomical_structure Intra articular Genetics medicine Iduronidase Molecular Biology Canine model |
Zdroj: | Molecular Genetics and Metabolism. 126:S149-S150 |
ISSN: | 1096-7192 |
Databáze: | OpenAIRE |
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