Partial correction of the Ξ±-galactosidas
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Malgorzata Przybylska; I-Huan Wu; Hongmei Zhao; Robin J. Ziegler; Jennifer D. To
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Article
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2004
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John Wiley and Sons
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English
β 163 KB
## Abstract ## Background Fabry disease is a recessive, Xβlinked disorder caused by a deficiency of the lysosomal enzyme Ξ±βgalactosidase A, leading to an accumulation of the glycosphingolipid globotriaosylceramide (GLβ3) in most tissues of the body. The goal of this study was to determine if syste