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Article

Effective cell and gene therapy in a murine model of Gaucher disease.

Proceedings of the National Academy of Sciences of the United States of America - 12 Sept 2006

Enquist Ida Berglin, Nilsson Eva, Ooka Andreas, Månsson Jan-Eric, Olsson Karin, Ehinger Mats, Brady Roscoe O, Richter Johan, Karlsson Stefan

Abstract excerpt

Gaucher disease (GD) is a lysosomal storage disorder due to an inherited deficiency in the enzyme glucosylceramidase (GCase) that causes hepatosplenomegaly, cytopenias, and bone disease as key clinical symptoms. Previous mouse models with GCase deficiency have been lethal in the perinatal period or viable without displaying the clinical features of GD. We have generated viable mice with characteristic clinical...

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Identifiers

PMID: 16954197
DOI: 10.1073/pnas.0606016103
PMCID: PMC1564262

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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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