Pompe disease: Current state of treatment modalities and animal models

Molecular Genetics and Metabolism

Volumn 92, Issue 4, 2007, Pages 299-307

  Geel, T M ^a   McLaughlin, P M J ^a   de Leij, L F M H ^a   Ruiters, M H J ^a   Niezen Koning, K E ^a  

^a UNIVERSITY OF GRONINGEN   (Netherlands)

Author keywords

Glucosidase; Enzyme replacement therapy; GAA; Gene correction therapy; Gene therapy; KO mouse models; Pompe disease

Indexed keywords

ADENOVIRUS VECTOR; GLUCAN 1,4 ALPHA GLUCOSIDASE; GLYCOGEN; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; UNCLASSIFIED DRUG;

AUTOSOMAL RECESSIVE DISORDER; CELL DAMAGE; DEATH; ENZYME ACTIVITY; ENZYME REPLACEMENT; GENE TARGETING; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; KNOCKOUT MOUSE; LYSOSOME STORAGE DISEASE; MULTIPLE ORGAN FAILURE; NONHUMAN; PRIORITY JOURNAL; REVIEW;

ALPHA-GLUCOSIDASES; ANIMALS; DISEASE MODELS, ANIMAL; GENE THERAPY; GLUCAN 1,4-ALPHA-GLUCOSIDASE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; MICE; MICE, KNOCKOUT; THERAPIES, INVESTIGATIONAL;

ANIMALIA;

EID: 36248964186     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2007.07.009     Document Type: Review

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