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Article

A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA.

EMBO molecular medicine - 1 May 2013

Sorrentino Nicolina Cristina, D'Orsi Luca, Sambri Irene, Nusco Edoardo, Monaco Ciro, Spampanato Carmine, Polishchuk Elena, Saccone Paola, De Leonibus Elvira, Ballabio Andrea, Fraldi Alessandro

Abstract excerpt

Mucopolysaccharidoses type IIIA (MPS-IIIA) is a neurodegenerative lysosomal storage disorder (LSD) caused by inherited defects of the sulphamidase gene. Here, we used a systemic gene transfer approach to demonstrate the therapeutic efficacy of a chimeric sulphamidase, which was engineered by adding the signal peptide (sp) from the highly secreted iduronate-2-sulphatase (IDS) and the blood-brain barrier...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 23568409
DOI: 10.1002/emmm.201202083
PMCID: PMC3662312

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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