Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

Abstract The pediatric lysosomal storage disorder mucopolysaccharidosis type II is caused by mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe neurodegeneration, skeletal disease, and cardiorespiratory disease. Most patients manifest with cognitive symptoms,...

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Principais autores:	Hélène FE Gleitz, Ai Yin Liao, James R Cook, Samuel F Rowlston, Gabriella MA Forte, Zelpha D'Souza, Claire O'Leary, Rebecca J Holley, Brian W Bigger
Formato:	Artigo
Idioma:	Inglês
Publicado:	Springer Nature 2018-06-01
Series:	EMBO Molecular Medicine
Assuntos:	apolipoprotein E blood–brain barrier Hunter mucopolysaccharidosis type II stem cell gene therapy
Acceso en liña:	https://doi.org/10.15252/emmm.201708730
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