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. 1987 Feb;84(4):906–909. doi: 10.1073/pnas.84.4.906

Complete correction of the enzymatic defect of type I Gaucher disease fibroblasts by retroviral-mediated gene transfer.

J Sorge, W Kuhl, C West, E Beutler
PMCID: PMC304328  PMID: 3547401

Abstract

Glucocerebrosidase cDNA and the neomycin-resistance gene (neo) were cloned into a retrovirus vector. Mouse fibroblasts infected with this vector expressed human glucocerebrosidase, which was readily distinguished from the mouse enzyme using mouse monoclonal anti-glucocerebrosidase antibodies. Cultured fibroblasts and transformed lymphoblasts from patients with type I Gaucher disease were infected with the retrovirus rescued from the mouse fibroblasts by a helper virus. Transformed cells were selected with the antibiotic G418. The enzyme activity of cells infected with virus containing glucocerebrosidase cDNA was restored to normal, while uninfected cells or cells infected with virus containing only the neo gene did not produce glucocerebrosidase.

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Selected References

These references are in PubMed. This may not be the complete list of references from this article.

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