Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 gaucher disease

PLoS ONE

Volumn 7, Issue 8, 2012, Pages

  Cabrera Salazar, Mario A ^a   DeRiso, Matthew ^a   Bercury, Scott D ^a   Li, Lingyun ^a   Lydon, John T ^a   Weber, William ^a   Pande, Nilesh ^a   Cromwell, Mandy A ^a   Copeland, Diane ^a   Leonard, John ^a   Cheng, Seng H ^a   Scheule, Ronald K ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CERAMIDE GLUCOSYLTRANSFERASE INHIBITOR; GZ 161; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD BRAIN BARRIER; BRAIN STEM; CEREBELLUM; CONTROLLED STUDY; ENZYME INHIBITION; ENZYME SUBSTRATE; FEMALE; GAUCHER DISEASE; HIPPOCAMPUS; LIFESPAN; LIPID BRAIN LEVEL; MOUSE; NEUROPATHOLOGY; NEWBORN; NONHUMAN; SURVIVAL; THALAMUS;

ANIMALS; BLOOD-BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; DNA PRIMERS; ENZYME INHIBITORS; GAUCHER DISEASE; GLUCOSYLCERAMIDES; GLUCOSYLTRANSFERASES; HISTOLOGICAL TECHNIQUES; INJECTIONS, INTRAPERITONEAL; KAPLAN-MEIER ESTIMATE; MICE; PSYCHOSINE;

EID: 84865084107     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0043310     Document Type: Article

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