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Article

Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction.

Neurobiology of disease - 1 Aug 2001

Luddi A, Volterrani M, Strazza M, Smorlesi A, Rafi M A, Datto J, Wenger D A, Costantino-Ceccarini E

Abstract excerpt

Galactocerebrosidase (GALC) is deficient in all tissues from human patients and animal models with globoid cell leukodystrophy (GLD) or Krabbe disease. The deficiency results in decreased lysosomal catabolism of certain galactolipids including galactosylceramide and psychosine that are synthesized maximally during myelination. According to current theories, the accumulation of psychosine in humans and animals...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Leukodystrophy, Globoid Cell

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Source

Identifiers

PMID: 11493025
DOI: 10.1006/nbdi.2001.0407

Genes

GALC
LAMP2

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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