Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-α-glucosidase

Proceedings of the National Academy of Sciences of the United States of America

Volumn 96, Issue 16, 1999, Pages 8861-8866

  Amalfitano, A ^a   Mcvie Wylie, A J ^a   Hu, H ^a   Dawson, T L ^a   Raben, N ^b   Plotz, P ^b   Chen, Y T ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b NATIONAL INSTITUTE OF ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES   (United States)

Author keywords

Gene therapy; Pompe

Indexed keywords

GLUCAN 1,4 ALPHA GLUCOSIDASE; VIRUS VECTOR;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; ENZYME ACTIVATION; GENE TARGETING; GENE THERAPY; GLYCOGEN ANALYSIS; GLYCOGEN METABOLISM; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUMAN CELL; INTRAVENOUS DRUG ADMINISTRATION; MOUSE; MUSCULAR DYSTROPHY; NONHUMAN; PRIORITY JOURNAL; TISSUE DISTRIBUTION;

ADENOVIRIDAE; ALPHA-GLUCOSIDASES; ANIMALS; CYTOMEGALOVIRUS; GENE THERAPY; GENES, POL; GENETIC VECTORS; GLYCOGEN; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; LIVER; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MUSCLE, SKELETAL; MUSCULAR DISEASES; PROMOTER REGIONS (GENETICS);

ADENOVIRIDAE; ANIMALIA;

EID: 0033529902     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.96.16.8861     Document Type: Article

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