Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy

Mucopolysaccharidosis type VI (MPS VI) is due to a deficiency of the lysosomal enzyme arylsulfatase B (ARSB) that results in multi-organ accumulation of glycosaminoglycans (GAGs). Limitations of current treatments prompted the development of a liver-directed gene therapy clinical trial for MPS VI. W...

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Veröffentlicht in:	Med (New York, N.Y. : Online) N.Y. : Online), 2024-11, p.100544, Article 100544
Hauptverfasser:	Rossi, Alessandro, Romano, Roberta, Fecarotta, Simona, Dell’Anno, Margherita, Pecorella, Valentina, Passeggio, Roberta, Zancan, Stefano, Parenti, Giancarlo, Santamaria, Francesca, Borgia, Francesco, Deodato, Federica, Funghini, Silvia, Rupar, Charles A., Prasad, Chitra, O’Callaghan, Mar, Mitchell, John J., Valsecchi, Maria Grazia, la Marca, Giancarlo, Galimberti, Stefania, Auricchio, Alberto, Brunetti-Pierri, Nicola
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV adeno-associated virus arylsulfatase B gene therapy inherited metabolic disorders lysosomal storage disorders mucopolysaccharidosis type VI
Online-Zugang:	Volltext
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