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Literature summary for 2.4.1.80 extracted from

  • McEachern, K.A.; Fung, J.; Komarnitsky, S.; Siegel, C.S.; Chuang, W.L.; Hutto, E.; Shayman, J.A.; Grabowski, G.A.; Aerts, J.M.; Cheng, S.H.; Copeland, D.P.; Marshall, J.
    A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease (2007), Mol. Genet. Metab., 91, 259-267.
    View publication on PubMed

Application

Application Comment Organism
medicine substrate inhibition therapy with Genz-112638 represents a viable alternate approach to enzyme therapy to treat the visceral pathology in Gaucher disease Mus musculus

Inhibitors

Inhibitors Comment Organism Structure
Genz-112638 inhibitor is tested in a murine model of Gaucher disease (D409V/null): mice that receive drug prior to significant accumulation of substrate show reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to control. Treatment of older mice that already display significant amounts of tissue glucosylceramide results in arrest of further accumulation of the substrate and appearance of additional Gaucher cells in affected organs; substrate inhibition therapy with Genz-112638 represents an approach to enzyme therapy to treat the visceral pathology in Gaucher diseases Mus musculus

Organism

Organism UniProt Comment Textmining
Mus musculus
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-
-

Synonyms

Synonyms Comment Organism
glucosylceramide synthase
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Mus musculus

IC50 Value

IC50 Value IC50 Value Maximum Comment Organism Inhibitor Structure
0.000024
-
-
Mus musculus Genz-112638