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Article

Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy.

Molecules and cells - 1 Jul 2010

Jung Sung-Chul, Park Eun-Sook, Choi Eun Nam, Kim Chi Hwa, Kim Su Jin, Jin Dong-Kyu

Abstract excerpt

Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked inherited disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS), which results in the lysosomal accumulation of glycosaminoglycans (GAG) such as dermatan and heparan sulfate. Here, we report the generation of IDS knockout mice, a model of human MPS II, and an analysis of the resulting phenotype. We also evaluated the effect...

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Source

Identifiers

PMID: 20652491
DOI: 10.1007/s10059-010-0083-2

Genes

IDS

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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