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Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice

Deposition of heparan sulfate (HS) in the brain of patients with mucopolysaccharidosis II (MPS II) is believed to be the leading cause of neurodegeneration, resulting in several neurological signs and symptoms, including neurocognitive impairment. We recently showed that pabinafusp alfa, a blood-bra...

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Detalles Bibliográficos
Autores principales:	Morimoto, Hideto, Morioka, Hiroki, Imakiire, Atsushi, Yamamoto, Ryuji, Hirato, Tohru, Sonoda, Hiroyuki, Minami, Kohtaro
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9142692/ https://www.ncbi.nlm.nih.gov/pubmed/35662814 http://dx.doi.org/10.1016/j.omtm.2022.05.002

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