AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice

Molecular Therapy

Volumn 10, Issue 1, 2004, Pages 106-116

  Hennig, Anne K ^a   Ogilvie, Judith Mosinger ^a   Ohlemiller, Kevin K ^a   Timmers, Adrian M ^b   Hauswirth, William W ^b   Sands, Mark S ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

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

Author keywords

Adeno associated virus vector; Electroretinography; Gene therapy; Lysosomal storage disease; Mucopolysaccharidosis; Retinal degeneration

Indexed keywords

BETA GLUCURONIDASE; GLYCOSAMINOGLYCAN; PARVOVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DISEASE ASSOCIATION; DOSE RESPONSE; DRUG EFFICACY; ELECTRORETINOGRAPHY; ENZYME ACTIVITY; GENE THERAPY; HUNTER SYNDROME; INJECTION SITE; LYSOSOME STORAGE DISEASE; MOUSE; NONHUMAN; PIGMENT EPITHELIUM; RETINA CELL; RETINA DEGENERATION; VISION;

ANIMALS; DEPENDOVIRUS; ELECTRORETINOGRAPHY; GENE THERAPY; GENETIC VECTORS; GLUCURONIDASE; GLYCOSAMINOGLYCANS; INJECTIONS; LYSOSOMES; MICE; MICE, MUTANT STRAINS; MUCOPOLYSACCHARIDOSIS VII; PIGMENT EPITHELIUM OF EYE; RETINA; TISSUE DISTRIBUTION; VITREOUS BODY;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 3042812075     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.03.018     Document Type: Article

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