The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of fabry disease

Molecular Therapy

Volumn 18, Issue 1, 2010, Pages 23-33

  Khanna, Richie ^a   Soska, Rebecca ^a   Lun, Yi ^a   Feng, Jessie ^a   Frascella, Michelle ^a   Young, Brandy ^a   Brignol, Nastry ^a   Pellegrino, Lee ^a   Sitaraman, Sheela A ^a   Desnick, Robert J ^b   Benjamin, Elfrida R ^a   Lockhart, David J ^a   Valenzano, Kenneth J ^a  

^a Amicus Therapeutics   (United States)

^b MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

1 DEOXYGALACTONOJIRIMYCIN; AGALSIDASE BETA; ALPHA GALACTOSIDASE; CHAPERONE; GLOBOTRIAOSYLCERAMIDE; MUTANT PROTEIN; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD LEVEL; BRAIN LEVEL; CONTROLLED STUDY; DOSE RESPONSE; DRUG EFFECT; DRUG MECHANISM; ENZYME ACTIVITY; ENZYME STABILITY; FABRY DISEASE; HEART; KIDNEY; MALE; MOUSE; MULTIPLE CYCLE TREATMENT; NONHUMAN; PROTEIN EXPRESSION; SKIN; TREATMENT DURATION;

1-DEOXYNOJIRIMYCIN; ALPHA-GALACTOSIDASE; ANIMALS; BLOTTING, WESTERN; DISEASE MODELS, ANIMAL; FABRY DISEASE; HUMANS; IMMUNOHISTOCHEMISTRY; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MICE, TRANSGENIC; TRIHEXOSYLCERAMIDES;

MUS; MUS MUSCULUS;

EID: 74149090458     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.220     Document Type: Article

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