Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy

Molecular Therapy

Volumn 6, Issue 6, 2002, Pages 745-758

  Xu, Lingfei ^a   Mango, Robert L ^a   Sands, Mark S ^a   Ellinwood, N Matthew ^b   Ponder, Katherine Parker ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Gene therapy; Glycosaminoglycans; Liver; Lysosomal storage disease; Mucopolysaccharidosis; Neonatal; Retroviral vector; glucuronidase

Indexed keywords

BETA GLUCURONIDASE; MANNOSE 6 PHOSPHATE; RETROVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; AORTA; BRAIN; BRONCHUS MUCOSA; CLINICAL FEATURE; CONTROLLED STUDY; CORNEA; DOG; ENZYME ACTIVITY; ENZYME BLOOD LEVEL; ENZYME DEFICIENCY; ENZYME RELEASE; GENETIC TRANSDUCTION; GLIA CELL; HEART VALVE; HISTOPATHOLOGY; KIDNEY; LIVER CELL; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NERVE CELL; NEWBORN; NEWBORN PERIOD; NONHUMAN; PIGMENT EPITHELIUM; PROTEIN EXPRESSION; REVIEW; SMALL INTESTINE; SPLEEN; THYMUS; VIRAL GENE THERAPY;

ANIMALIA; BRONCHUS; CANIS FAMILIARIS; UNIDENTIFIED RETROVIRUS;

EID: 0036932904     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0809     Document Type: Review

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