A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease

Molecular Genetics and Metabolism

Volumn 91, Issue 3, 2007, Pages 259-267

  McEachern, Kerry Anne ^a   Fung, John ^a   Komarnitsky, Svetlana ^a   Siegel, Craig S ^a   Chuang, Wei Lien ^a   Hutto, Elizabeth ^a   Shayman, James A ^b   Grabowski, Gregory A ^c   Aerts, Johannes M F G ^d   Cheng, Seng H ^a   Copeland, Diane P ^a   Marshall, John ^a  

^a GENZYME CORPORATION   (United States)

^b UNIVERSITY OF MICHIGAN   (United States)

^c CINCINNATI CHILDREN S HOSPITAL RESEARCH FOUNDATION   (United States)

^d UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

Gangliosides; Gaucher disease; Glucocerebrosidase; Glucosylceramide; Glycosphingolipids; Lysosomal storage disorders; Substrate inhibition therapy

Indexed keywords

CERAMIDE GLUCOSYLTRANSFERASE INHIBITOR; GENZ 112638; GLUCOSYLCERAMIDE; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DRUG DOSE INCREASE; DRUG POTENCY; DRUG SELECTIVITY; ENZYME INHIBITION; ENZYME SUBSTRATE; FEMALE; GAUCHER DISEASE; HUMAN; HUMAN CELL; IC 50; LIVER; LUNG; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SINGLE DRUG DOSE; SPLEEN;

AGE FACTORS; ANIMALS; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDES; GLUCOSYLTRANSFERASES; GLYCOSPHINGOLIPIDS; MICE; MICE, KNOCKOUT; PYRROLIDINES; SUBSTRATE SPECIFICITY;

ANIMALIA; MURINAE; MUS;

EID: 34249306894     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2007.04.001     Document Type: Article

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