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Article

Expression of human glucocerebrosidase in murine macrophages: identification of efficient retroviral vectors.

Experimental hematology - 1 Aug 1994

Freas-Lutz D L, Correll P H, Dougherty S F, Xu L, Pluznik D H, Karlsson S

Abstract excerpt

Gaucher's disease is an autosomal recessive disorder characterized by a functional deficiency in beta-glucocerebrosidase enzymatic activity and the resultant accumulation of the glycolipid glucocerebroside in macrophages. Due to the nature of the affected cells, Gaucher's disease is an excellent candidate for gene therapy of hematopoietic stem cells and autologous bone marrow transplantation of transduced cells...

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PMID: 8062885

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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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