GLUCOCEREBROSIDASE GENE TRANSFER TO CD34 CELLS

$0M01FY2000RRNIH

University Of Pittsburgh At Pittsburgh, Pittsburgh PA

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Abstract

Gaucher disease results from inherited mutations in the gene for the lysosomal enzyme glucocerebrosidase (GC). Patients suffer from hepatosplenomegaly proceeding to hepatic failure, bone deterioration with multiple fractures and in some patients, progressive neurological degeneration. The symptoms in Gaucher disease are a consequence of the accumulation of the lipid substrate for glucocerebrosidase in lysosomes of tissue macrophages. Allogeneic bone marrow transplantation results in the replacement of enzyme deficient by normal ones.

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