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Article

Preselective gene therapy for Fabry disease

6 Mar 2001

Abstract excerpt

Fabry disease is a lipid storage disorder resulting from mutations in the gene encoding the enzyme alpha-galactosidase A (alpha-gal A; EC ). We previously have demonstrated long-term alpha-gal A enzyme correction and lipid reduction mediated by therapeutic ex vivo transduction and transplantation of hematopoietic cells in a mouse model of Fabry disease. We now report marked improvement in the efficiency of this...

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Identifiers

PMID: 11248095
DOI: 10.1073/pnas.061020598

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Source receipt: pubmed, retrieved 7 Sept 2026.

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