Enhanced secretion and uptake of β-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice

Molecular Therapy

Volumn 5, Issue 5, 2002, Pages 617-626

  Elliger, Susan S ^a   Elliger, Carl A ^a   Lang, Chen ^a   Watson, Gordon L ^a  

^a CHILDREN S HOSPITAL OAKLAND RESEARCH INSTITUTE   (United States)

Author keywords

AAV; Adeno associated virus; Intrathecal; Lysosomal storage disease; MPS VII; Mucopolysaccharidosis; Protein transduction domain; Secretion signal; glucuronidase

Indexed keywords

AMINO ACID; BETA GLUCURONIDASE; ESTERASE; GLYCOSAMINOGLYCAN; IMMUNOGLOBULIN KAPPA CHAIN; LYSOSOME ENZYME; MANNOSE 6 PHOSPHATE; MATERNAL ANTIBODY; RNA; SOMATOMEDIN B RECEPTOR; TRANSACTIVATOR PROTEIN; VIRUS DNA;

ADENO ASSOCIATED VIRUS; ANIMAL BEHAVIOR; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE GROWTH; BRAIN TISSUE; BREEDING; CONTROLLED STUDY; DRUG ADMINISTRATION ROUTE; DRUG BLOOD LEVEL; DRUG TISSUE LEVEL; ENZYME LOCALIZATION; ENZYME MODIFICATION; ENZYME RELEASE; FEMALE; GENE SEQUENCE; GENE THERAPY; GENETIC CODE; GROWTH INHIBITION; HEART; HISTOPATHOLOGY; LIVER; MALE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NEWBORN; NONHUMAN; PHENOTYPE; SPLEEN; VIRAL GENE DELIVERY SYSTEM;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 0036098944     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0594     Document Type: Article

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