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Article

Beta-galactosidase deficiency: an approach to chaperone therapy.

Journal of inherited metabolic disease - 1 Jan 2000

Suzuki Yoshiyuki

Abstract excerpt

We propose a new molecular therapeutic approach to lysosomal diseases with severe neurological manifestations. Some low-molecular-weight compounds, acting as competitive inhibitors of a lysosomal enzyme in vitro, were found to stabilize and restore catalytic activities of the enzyme molecule as a molecular chaperone. We started this trial first in Fabry disease (generalized vasculopathy) using galactose and...

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Share to beta-Galactosidase
Share to Dose-Response Relationship, Drug
Share to Enzyme Induction

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to G(M1) Ganglioside

Share to Gangliosidosis, GM1

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Source

Identifiers

PMID: 16763919
DOI: 10.1007/s10545-006-0287-y

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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