Title : Correction of acid beta-galactosidase deficiency in GM1 gangliosidosis human fibroblasts by retrovirus vector-mediated gene transfer: higher efficiency of release and cross-correction by the murine enzyme.

Pub. Date : 2000 Mar 20

PMID : 10757351






2 Functional Relationships(s)
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1 Mutations in the lysosomal acid beta-galactosidase (EC 3.2.1.23) underlie two different disorders: GM1 gangliosidosis, which involves the nervous system and visceral organs to varying extents, and Morquio"s syndrome type B (Morquio B disease), which is a skeletal-connective tissue disease without any CNS symptoms. G(M1) Ganglioside galactosidase beta 1 Homo sapiens
2 This article shows that transduction of human GM1 gangliosidosis fibroblasts with retrovirus vectors encoding the human acid beta-galactosidase cDNA leads to complete correction of the enzymatic deficiency. G(M1) Ganglioside galactosidase beta 1 Homo sapiens