Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?

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作者： Shunji,Tomatsu ^[1] ; Adriana M,Montaño ^[2] ; Hirotaka,Oikawa ^[3] ; Vu Chi,Dung ^[4] ; Amiko,Hashimoto ^[5] ; Toshihiro,Oguma ^[6] ; Monica L,Gutiérrez ^[7] ; Tatsuo,Takahashi ^[8] ; Tsutomu,Shimada ^[9] ; Tadao,Orii ^[10] ; William S,Sly ^[11]

作者单位： Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA. Electronic address: stomatsu@nemours.org. ^[1] Department of Pediatrics, Saint Louis University Doisy Research Center, St. Louis, MO, USA; Department of Biochemistry and Molecular Biology, Saint Louis University School of Medicine, St. Louis, MO, USA. ^[2] Suzuka University of Medical Science, School of Pharmacy, Japan. ^[3] Department of Endocrinology, Metabolism & Genetics, Vietnam National Hospital of Pediatrics, Hanoi, Viet Nam. ^[4] Myojinshita Aoki Dermatology Clinic, Tokyo, Japan. ^[5] Daiichi-Sankyo Pharmaceutical Co., Tokyo, Japan. ^[6] Department of Pediatrics, Saint Louis University Doisy Research Center, St. Louis, MO, USA. ^[7] Department of Clinical Pharmacy, Faculty of Pharmaceutical Sciences, Hokuriku University, Kanazawa, Japan. ^[8] Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA. ^[9] Department of Pediatrics, Gifu University, School of Medicine, Gifu, Japan. ^[10] Department of Biochemistry and Molecular Biology, Saint Louis University School of Medicine, St. Louis, MO, USA. ^[11]

关键词 Enzyme replacement therapy Fibrous cartilage Hyaline cartilage Mucopolysaccharidosis IVA N-acetylgalactosamine-6-sulfate sulfatase