Intercellular transfer of the virally derived precursor form of acid α-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy pompe disease

Human Gene Therapy

Volumn 12, Issue 5, 2001, Pages 527-538

  Pauly, D F ^a,b   Fraites, T J ^b   Toma, C ^a   Bayes, H S ^a   Huie, M L ^c   Hirschhorn, R ^c   Plotz, P H ^d   Raben, N ^d   Kessler, P D ^a   Byrne, B J ^a,b  

^a UNIVERSITY OF FLORIDA   (United States)

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

^c NEW YORK UNIVERSITY MEDICAL CENTER   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

GLUCAN 1,4 ALPHA GLUCOSIDASE; SOMATOMEDIN B RECEPTOR; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CARDIOMYOPATHY; ENZYME DEFICIENCY; GENE THERAPY; GENE TRANSFER; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUMAN CELL; IMMUNITY; IMMUNOCYTOCHEMISTRY; MOUSE; NONHUMAN; PROTEIN EXPRESSION; WESTERN BLOTTING;

ADENOVIRIDAE; ALPHA-GLUCOSIDASES; ANIMALS; BLOTTING, WESTERN; CELLS, CULTURED; COCULTURE TECHNIQUES; DNA, COMPLEMENTARY; FIBROBLASTS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNOHISTOCHEMISTRY; LYSOSOMES; MANNOSEPHOSPHATES; MICE; MICE, KNOCKOUT; MICE, NUDE; MUSCLE, SKELETAL; MYOCARDIUM; PLACENTA; RECEPTOR, IGF TYPE 2; RECOMBINANT PROTEINS; TIME FACTORS; TRANSDUCTION, GENETIC;

ADENOVIRIDAE; ANIMALIA; MURINAE;

EID: 0035937315     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/104303401300042447     Document Type: Article

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