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Published in 2019 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms20082014
Abstract: Mucopolysaccharidosis type II (MPSII) is a lysosomal storage disorder due to the deficit of the enzyme iduronate 2-sulfatase (IDS), which leads to the accumulation of glycosaminoglycans in most organ-systems, including the brain, and resulting in…
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Keywords:
mpsii preclinical;
enzyme;
targeting brain;
brain disease ... See more keywords