Article
Correction of a lysosomal deficiency by contact-mediated enzyme transfer after bone marrow transplantation.
Transplantation - 1 Oct 1993
Bou-Gharios G, Adams G, Pace P, Warden P, Olsen I
Abstract excerpt
The effectiveness of bone marrow transplantation for treating lysosomal deficiency diseases relies on the ability of bone marrow cells to provide the missing enzyme to various tissues of the recipient. This has been shown to occur in vitro by endocytosis of enzyme secreted by bone marrow-derived cells and also by direct cell-to-cell-contact. To investigate the mechanism of enzyme replacement therapy in vivo we...
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