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Article

Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2015

Dahl Maria, Doyle Alexander, Olsson Karin, Månsson Jan-Eric, Marques André R A, Mirzaian Mina, Aerts Johannes M, Ehinger Mats, Rothe Michael, Modlich Ute, Schambach Axel, Karlsson Stefan

Abstract excerpt

Gaucher disease is caused by an inherited deficiency of the enzyme glucosylceramidase. Due to the lack of a fully functional enzyme, there is progressive build-up of the lipid component glucosylceramide. Insufficient glucosylceramidase activity results in hepatosplenomegaly, cytopenias, and bone...

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Share to Antigens, Differentiation, Myelomonocytic
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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: 25655314
DOI: 10.1038/mt.2015.16
PMCID: PMC4427872

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-25655314-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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