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

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, which ca...

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Detalhes bibliográficos
Publicado no:EMBO Mol Med
Main Authors: Gleitz, Hélène FE, Liao, Ai Yin, Cook, James R, Rowlston, Samuel F, Forte, Gabriella MA, D'Souza, Zelpha, O'Leary, Claire, Holley, Rebecca J, Bigger, Brian W
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2018
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6034129/
https://ncbi.nlm.nih.gov/pubmed/29884617
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201708730
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