Complete correction of acid α-glucosidase deficiency in Pompe disease fibroblasts in vitro, and lysosomally targeted expression in neonatal rat cardiac and skeletal muscle

Gene Therapy

Volumn 5, Issue 4, 1998, Pages 473-480

  Pauly, D F ^a   Johns, D C ^a   Matelis, L A ^a   Lawrence, J H ^a   Byrne, B J ^a,c   Kessler, P D ^a,b  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b Ross Research Building ^*  (United States)

^c UNIVERSITY OF FLORIDA   (United States)

Author keywords

Adenovirus; Lysosomes; Myopathy; Storage disease

Indexed keywords

GLUCAN 1,4 ALPHA GLUCOSIDASE; GLYCOGEN; RECOMBINANT PROTEIN;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; ENZYME ACTIVITY; ENZYME DEFICIENCY; FIBROBLAST; GENE EXPRESSION; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HEART MUSCLE CELL; HUMAN; HUMAN CELL; IMMUNOBLOTTING; LYSOSOME; NEWBORN; NONHUMAN; PRIORITY JOURNAL; RAT; SKELETAL MUSCLE; VIRUS RECOMBINANT;

ADENOVIRIDAE; ANIMALIA;

EID: 0031942298     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300609     Document Type: Article

References (48)

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