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Enhanced Version of Human Sulfamidase Significantly Ameliorates CNS Pathology When Delivered to the MPS-IIIA Mice by AAV-Mediated Intra-CSF Injection

Document Type

Journal

Author

Sorrentino, Nicolina C.; Cacace, Vincenzo; Maffia, Veronica; Strollo, Sandra; Tedesco, Novella; Nusco, Edoardo; Romagnoli, Noemi; Ventrella, Domenico; Barone, Francesca; Bacci, Marialaura; Huang, Yan; Liu, Nan; Kalled, Susan; Choi, Vivian; Fraldi, Alessandro

Source

MOLECULAR THERAPY; MAY 2018, 26 5, p132-p132, 1p. Supplement: 1

Subject

Language

English

ISSN

15250024

Online Access

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