Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression

Journal of Gene Medicine

Volumn 11, Issue 10, 2009, Pages 913-920

  Sun, Baodong ^a   Zhang, Haoyue ^a   Bird, Andrew ^a   Li, Songtao ^a   Young, Sarah P ^a   Koeber, Dwight D ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Acid alpha glucosidase; Acid maltase; Adeno associated virus; Glycogen storage disease type II; Pompe disease

Indexed keywords

GLUCAN 1,4 ALPHA GLUCOSIDASE; GLYCOGEN; LYSOSOME ASSOCIATED MEMBRANE PROTEIN 2; PARVOVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIOCHEMISTRY; CONTROLLED STUDY; DRUG EFFICACY; EARLY INTERVENTION; EMBRYO; ENZYME ACTIVITY; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; GLYCOGEN STORAGE DISEASE TYPE 2; HEART; HUMAN; HUMAN CELL; LYSOSOME; MOUSE; NONHUMAN; PRIORITY JOURNAL; ROTAROD TEST; SKELETAL MUSCLE; TISSUE SPECIFICITY; TRANSGENE; URINALYSIS; VIRAL GENE DELIVERY SYSTEM; WESTERN BLOTTING;

AGE FACTORS; ALPHA-GLUCOSIDASES; ANIMALS; DEPENDOVIRUS; ENZYME REPLACEMENT THERAPY; FEMALE; GENETIC VECTORS; GLYCOGEN; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; LIVER; LYSOSOMES; MALE; MICE; MUSCLES; SEX FACTORS; TRANSGENES;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 72449209826     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1372     Document Type: Article

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