Reversal of advanced disease in lysosomal acid lipase deficient mice: A model for lysosomal acid lipase deficiency disease

Molecular Genetics and Metabolism

Volumn 112, Issue 3, 2014, Pages 229-241

  Sun, Ying ^a,b   Xu, You Hai ^a,b   Du, Hong ^c   Quinn, Brian ^a   Liou, Benjamin ^a   Stanton, Lori ^a   Inskeep, Venette ^a   Ran, Huimin ^a   Jakubowitz, Phillip ^a,d   Grilliot, Nicholas ^a,d   Grabowski, Gregory A ^a,b,e  

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

^b UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

^c INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF CINCINNATI   (United States)

^e Synageva BioPharma Corp   (United States)

Author keywords

Cholesterol esters; Enzyme therapy; Lysosomal acid lipase; Lysosome; Triglycerides

Indexed keywords

ACID LIPASE; CHOLESTEROL ESTER; RECOMBINANT ENZYME; RECOMBINANT LYSOSOMAL ACID LIPASE; TRIACYLGLYCEROL; UNCLASSIFIED DRUG; CHOLESTEROL ESTERASE; LIPID;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL PROLIFERATION; CONTROLLED STUDY; ENZYME THERAPY; FEMALE; GENE MUTATION; HEPATOSPLENOMEGALY; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HYDROLYSIS; LIFESPAN; LIPA GENE; LIVER FIBROSIS; LIVER SIZE; LYSOSOME; MACROPHAGE; MALE; MOUSE; MUTATOR GENE; NONHUMAN; PRIORITY JOURNAL; SMALL INTESTINE; SPLEEN SIZE; SURVIVAL; WOLMAN DISEASE; ANIMAL; BLOOD; BODY WEIGHT; DISEASE MODEL; ENZYME REPLACEMENT; GENETICS; KNOCKOUT MOUSE; LIPID METABOLISM; METABOLISM; MORTALITY; ORGAN SIZE; PATHOLOGY; PHENOTYPE; TREATMENT OUTCOME;

MUS;

ANIMALS; BODY WEIGHT; DISEASE MODELS, ANIMAL; ENZYME REPLACEMENT THERAPY; FEMALE; LIPID METABOLISM; LIPIDS; MALE; MICE; MICE, KNOCKOUT; ORGAN SIZE; PHENOTYPE; STEROL ESTERASE; TREATMENT OUTCOME; WOLMAN DISEASE;

EID: 84902549916     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2014.04.006     Document Type: Article

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