Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease

Neurobiology of Disease

Volumn 16, Issue 3, 2004, Pages 506-515

  Andersson, Ulrika ^a   Smith, David ^a   Jeyakumar, Mylvaganam ^a   Butters, Terry D ^a   Borja, Mario Cortina ^b   Dwek, Raymond A ^a   Platt, Frances M ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

BMT; bone marrow transplantation; CNS; Gangliosidosis; glycosphingolipid; GSL; Imino sugar; N butyldeoxygalactonojirimycin; N butyldeoxynojirimycin; NB DGJ; NB DNJ; Sandhoff disease; SRT; substrate reduction therapy; Substrate reduction therapy

Indexed keywords

GALACTOSE; GLUCOSYLTRANSFERASE; GLYCOSIDASE; GLYCOSPHINGOLIPID; MIGLUSTAT; N BUTYLDEOXYGALACTONOJIRIMYCIN; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG SCREENING; ENZYME INHIBITION; FEMALE; GANGLIOSIDOSIS; GASTROINTESTINAL DISEASE; LIFE EXPECTANCY; LIPOGENESIS; MOUSE; NONHUMAN; PRIORITY JOURNAL; SANDHOFF DISEASE; SYMPTOM; TREATMENT OUTCOME; WEIGHT REDUCTION;

1-DEOXYNOJIRIMYCIN; ANIMALS; BEHAVIOR, ANIMAL; BETA-N-ACETYLHEXOSAMINIDASE; BODY WEIGHT; BRAIN; DISEASE MODELS, ANIMAL; FEMALE; GANGLIOSIDES; LIFE EXPECTANCY; LIVER; MALE; MICE; MICE, MUTANT STRAINS; SANDHOFF DISEASE;

EID: 3242681806     PISSN: 09699961     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nbd.2004.04.012     Document Type: Article

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