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Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA.

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第一作者： Nicolina Cristina,Sorrentino

第一单位： Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, Pozzuoli, Naples, Italy.

作者： Nicolina Cristina,Sorrentino ^[1] ; Vincenzo,Cacace ^[1] ; Maria,De Risi ^[1] ; Veronica,Maffia ^[1] ; Sandra,Strollo ^[1] ; Novella,Tedesco ^[1] ; Edoardo,Nusco ^[1] ; Noemi,Romagnoli ^[2] ; Domenico,Ventrella ^[2] ; Yan,Huang ^[3] ; Nan,Liu ^[3] ; Susan L,Kalled ^[3] ; Vivian W,Choi ^[3] ; Elvira,De Leonibus ^[4] ; Alessandro,Fraldi ^[5]

作者单位： Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, Pozzuoli, Naples, Italy. ^[1] Department of Veterinary Medical Sciences, University of Bologna, Via Tolara di Sopra 50, Ozzano dell'Emilia, Bologna, Italy. ^[2] Takeda Pharmaceuticals, Cambridge, MA, USA. ^[3] Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, Pozzuoli, Naples, Italy.;Institute of Cellular Biology and Neurobiology (IBCN), National Research Council (CNR), Via Ramarini 32, Monterotondo, Rome, Italy. ^[4] Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, Pozzuoli, Naples, Italy.;Department of Translational Medicine, University of Naples "Federico II," Naples, Italy. ^[5]

DOI 10.1016/j.omtm.2019.10.009

PMID 31788497

发布时间 2024-07-23

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Molecular therapy. Methods & clinical development

2019年15卷

333-342页

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Molecular therapy. Methods & clinical development

2019年15卷

333-342页

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Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA.

Molecular therapy. Methods & clinical development

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Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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