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Feasibility of gene therapy in Gaucher disease using an adeno-associated virus vector

Title
Feasibility of gene therapy in Gaucher disease using an adeno-associated virus vector
Authors
Hong Y.B.Kim E.Y.Yoo H.-W.Jung S.-C.
Ewha Authors
정성철
SCOPUS Author ID
정성철scopus
Issue Date
2004
Journal Title
Journal of Human Genetics
ISSN
1434-5161JCR Link
Citation
Journal of Human Genetics vol. 49, no. 10, pp. 536 - 543
Indexed
SCI; SCIE; SCOPUS WOS scopus
Document Type
Article
Abstract
Gaucher disease, one of the common lysosomal storage disorders, is caused by a deficiency of glucocerebrosidase (GC). We investigated gene transfer using recombinant adeno-associated viral (rAAV) vectors containing human GC cDNA driven by the human elongation factor 1-α promoter. This rAAV vector mediated efficient expression of human GC in human Gaucher fibroblasts. GC activities were increased from 2.8 to 3.4 times in normal fibroblast and from 1.9 to 4.6 times in Gaucher fibroblasts, and these increases in GC activity were maintained over 20 weeks. Intravenous administration of vectors via the hepatic portal vein and tail vein of wild-type mice resulted in efficient transduction into the tissues. GC activities of the liver, spleen, and lung in transduced mice were increased significantly up to two fold at 6 weeks after transduction. Significantly increased GC activities persisted over 20 weeks. Therefore, rAAV vector-mediated gene transfer may provide a therapeutic approach for the treatment of Gaucher disease. © The Japan Society of Human Genetics and Springer-Verlag 2004.
DOI
10.1007/s10038-004-0186-8
Appears in Collections:
의과대학 > 의학과 > Journal papers
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