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Article

Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2017

Fu Haiyan, Meadows Aaron S, Ware Tierra, Mohney Robert P, McCarty Douglas M

Abstract excerpt

Mucopolysaccharidosis (MPS) IIIB is a lysosomal storage disease with complex CNS and somatic pathology due to a deficiency in α-N-acetylglucosaminidase (NAGLU). Using global metabolic profiling by mass spectrometry targeting 361 metabolites, this study detected significant decreases in 225 and increases in six metabolites in serum samples from 7-month-old MPS IIIB mice, compared to wild-type (WT) mice. The...

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Source

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Identifiers

PMID: 28143737
DOI: 10.1016/j.ymthe.2016.12.025
PMCID: PMC5363204

Genes

NAGLU

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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