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Genetics and Gene Therapy in Hunter Disease.

Current gene therapy - 1 Jan 2018

Sestito S, Falvo F, Scozzafava C, Apa R, Pensabene L, Bonapace G, Moricca M T, Concolino D

Abstract excerpt

Mucopolysaccharidosis type II or Hunter syndrome is an X-linked lysosomal storage disease caused by a mutation in the gene encoding the lysosomal enzyme iduronate-2-sulfatase. The consequent enzyme deficiency causes a progressive, multisystem accumulation of glycosaminoglycans, which is the cause of the clinical manifestations involving also Central Nervous System for patients with the severe form of disease. The...

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Source

Identifiers

PMID: 29618310
DOI: 10.2174/1566523218666180404155759

Genes

IDS

Provenance

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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