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Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA

OBJECTIVE: Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease‐modifying therapy is available. The AAVance gene therapy trial investigates AAVrh.10 overexpressing human sulfamidase (LYS...

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Detalles Bibliográficos
Autores principales:	Bugiani, Marianna, Abbink, Truus E. M., Edridge, Arthur W. D., van der Hoek, Lia, Hillen, Anne E. J., van Til, Niek P., Hu‐A‐Ng, Gino V., Breur, Marjolein, Aiach, Karen, Drevot, Philippe, Hocquemiller, Michaël, Laufer, Ralph, Wijburg, Frits A., van der Knaap, Marjo S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2023
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10270249/ https://www.ncbi.nlm.nih.gov/pubmed/37165777 http://dx.doi.org/10.1002/acn3.51772

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