Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice

PLoS ONE

Volumn 5, Issue 8, 2010, Pages

  Dvorak Ewell, Melita ^a   Wendt, Dan ^a   Hague, Chuck ^a   Christianson, Terri ^a   Koppaka, Vish ^a   Crippen, Danielle ^b   Kakkis, Emil ^a   Vellard, Michel ^a  

^a BIOMARIN PHARMACEUTICAL INC   (United States)

^b BUCK INSTITUTE FOR RESEARCH ON AGING   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGGRECAN; COLLAGEN TYPE 1; COLLAGEN TYPE 10; COLLAGEN TYPE 2; KERATAN SULFATE; N ACETYLGALACTOSAMINE 6 SULFATASE; RECOMBINANT N ACETYLGALACTOSAMINE 6 SULFATASE; SOMATOMEDIN B RECEPTOR; TRANSCRIPTION FACTOR SOX9; UNCLASSIFIED DRUG; GALNS PROTEIN, HUMAN; N ACETYLGALACTOSAMINE 4 SULFATASE; RECOMBINANT PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CARTILAGE; CARTILAGE CELL; CHO CELL; CONTROLLED STUDY; DOSE RESPONSE; DRUG DISTRIBUTION; DRUG MECHANISM; DRUG PURIFICATION; DRUG SYNTHESIS; ENZYME ACTIVITY; ENZYME PHOSPHORYLATION; ENZYME REPLACEMENT; FEMALE; FIBROBLAST; GENE EXPRESSION REGULATION; GLYCOSAMINOGLYCAN METABOLISM; GROWTH PLATE; HEART VALVE; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO STUDY; LYSOSOME; MORQUIO SYNDROME; MOUSE; NONHUMAN; TISSUE DISTRIBUTION; WILD TYPE; ANIMAL; CATTLE; DRUG EFFECT; ISOLATION AND PURIFICATION; LIVER; MACROPHAGE; METABOLISM; METHODOLOGY; PATHOLOGY; TRANSPORT AT THE CELLULAR LEVEL;

ANIMALIA; CRICETULUS GRISEUS; IVA; MUS;

ANIMALS; BIOLOGICAL TRANSPORT; CARTILAGE; CATTLE; CHONDROCYTES; CHONDROITINSULFATASES; ENZYME REPLACEMENT THERAPY; FEMALE; GENE EXPRESSION REGULATION; GROWTH PLATE; HEART VALVES; HUMANS; KERATAN SULFATE; LIVER; LYSOSOMES; MACROPHAGES; MICE; MUCOPOLYSACCHARIDOSIS IV; RECOMBINANT PROTEINS;

EID: 77957870094     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0012194     Document Type: Article

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