Fabry Disease

Reports from Ewha Womans University highlight recent research in fabry disease

Published in Gene Therapy Weekly, October 15th, 2009

According to recent research from Seoul, South Korea, "Fabry disease is a lysosomal storage disease caused by a deficiency of alpha-galactosidase A, which results in aberrant glycosphingolipid metabolism and accumulation of globotriaosylceramide (Gb3). Since a correlation between the level of Gb3 and clinical manifestations of Fabry disease has not been observed, we investigated potential diagnostic biomarkers."

"Hepatic and renal gene expression of male alpha-galactosidase A-deficient mice (Fabry mice) was compared with that of wildtype mice. Microarray analyses were performed using samples taken before and after intravenous infusion of alpha-galactosidase A. The...

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Source: Gene Therapy Weekly (2009-10-15)

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