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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Bibliographic Details

Published in:	EMBO Molecular Medicine
Main Authors:	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
Format:	Article
Language:	English
Published:	Springer Nature 2018-06-01
Subjects:	apolipoprotein E blood–brain barrier Hunter mucopolysaccharidosis type II stem cell gene therapy
Online Access:	https://doi.org/10.15252/emmm.201708730