Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice

Proceedings of the National Academy of Sciences of the United States of America

Volumn 101, Issue 9, 2004, Pages 3083-3088

  LeBowitz, Jonathan H ^a   Grubb, Jeffrey H ^b   Maga, John A ^a   Schmiel, Deborah H ^a   Vogler, Carole ^b   Sly, William S ^b  

^a Symbiontics Inc   (United States)

^b SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

glucuronidase; Enzyme replacement therapy; IGF II Man6 P receptor; Lysosomal storage disease; Receptor mediated endocytosis

Indexed keywords

BETA GLUCURONIDASE; CARBOHYDRATE; CELL SURFACE RECEPTOR; CHIMERIC PROTEIN; MANNOSE; MANNOSE 6 PHOSPHATE; RECOMBINANT PROTEIN; SOMATOMEDIN B; SOMATOMEDIN B RECEPTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL SURFACE; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; DRUG TARGETING; DRUG UPTAKE; ENZYME LOCALIZATION; ENZYME REPLACEMENT; FIBROBLAST; GLYCOSYLATION; GLYCOSYLATION INDEPENDENT LYSOSOMAL TARGETING; LYSOSOME; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; OSTEOBLAST; PODOCYTE; PRIORITY JOURNAL; PROTEIN TRANSPORT; TISSUE DISTRIBUTION;

ANIMALS; CHO CELLS; CRICETINAE; DISEASE MODELS, ANIMAL; ENZYMES; GENE THERAPY; GLUCURONIDASE; HUMANS; INSULIN-LIKE GROWTH FACTOR II; LYSOSOMES; MICE; MUCOPOLYSACCHARIDOSIS VII; MUTAGENESIS, INSERTIONAL; RECOMBINANT PROTEINS; TRANSFECTION;

ANIMALIA; MURINAE;

EID: 1542357668     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0308728100     Document Type: Article

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