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Article

Chaperone effect of sulfated disaccharide from heparin on mutant iduronate-2-sulfatase in mucopolysaccharidosis type II.

Molecular genetics and metabolism - 1 Feb 2018

Hoshina Hiroo, Shimada Yohta, Higuchi Takashi, Kobayashi Hiroshi, Ida Hiroyuki, Ohashi Toya

Abstract excerpt

Small molecules called pharmacological chaperones have been shown to improve the stability, intracellular localization, and function of mutated enzymes in several lysosomal storage diseases, and proposed as promising therapeutic agents for them. However, a chaperone compound for mucopolysaccharidosis type II (MPS II), which is an X-linked lysosomal storage disorder characterized by a deficiency of...

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Source

Identifiers

PMID: 29289480
DOI: 10.1016/j.ymgme.2017.12.428

Genes

IDS

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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