Sustained production of β-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice

Experimental Neurology

Volumn 160, Issue 1, 1999, Pages 17-27

  Skorupa, Annette F ^a   Fisher, Krisna J ^b   Wilson, James M ^b   Parente, Michael K ^a   Wolfe, John H ^a,b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

AAV vector; Animal model; CNS; Gene therapy; Lysosomal storage; Mucopolysaccharidoses; glucuronidase

Indexed keywords

BETA GLUCURONIDASE; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DEGENERATIVE DISEASE; ENZYME ACTIVITY; ENZYME DEFICIENCY; ENZYME LOCALIZATION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; IMMUNOHISTOCHEMISTRY; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN INDUCTION;

EID: 0032758906     PISSN: 00144886     EISSN: None     Source Type: Journal    
DOI: 10.1006/exnr.1999.7176     Document Type: Article

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