Infusion of recombinant human acid sphingomyelinase into Niemann-Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology

FASEB Journal

Volumn 14, Issue 13, 2000, Pages 1988-1995

  Miranda, Silvia R P ^a   He, Xingxuan ^a   Simonaro, Calogera M ^a   Gatt, Shimon ^b   Dagan, Arie ^b   Desnick, Robert J ^a   Schuchman, Edward H ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b HEBREW UNIVERSITY OF JERUSALEM   (Israel)

Author keywords

Animal models; Enzyme therapy; Lysosomal storage disease

Indexed keywords

SPHINGOMYELIN PHOSPHODIESTERASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY PRODUCTION; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; DRUG EFFICACY; DRUG INFUSION; ENZYME REPLACEMENT; MOUSE; NERVE DEGENERATION; NIEMANN PICK DISEASE; NONHUMAN; PATHOPHYSIOLOGY; PRIORITY JOURNAL; TREATMENT OUTCOME; WEIGHT GAIN;

ANIMALIA; CRICETINAE; CRICETULUS GRISEUS;

EID: 0033810516     PISSN: 08926638     EISSN: None     Source Type: Journal    
DOI: 10.1096/fj.00-0014com     Document Type: Article

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