Title : Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.

Pub. Date : 2018 Jul

PMID : 29884617






1 Functional Relationships(s)
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1 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. Dermatan Sulfate iduronate 2-sulfatase Homo sapiens