Title : Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.

Pub. Date : 2004 Jan

PMID : 14716680






1 Functional Relationships(s)
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1 BACKGROUND: Fabry disease is a recessive, X-linked disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A, leading to an accumulation of the glycosphingolipid globotriaosylceramide (GL-3) in most tissues of the body. globotriaosylceramide galactosidase, alpha Mus musculus