Article
Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.
Gene therapy - 1 Nov 2004
Xu F, Ding E, Liao S X, Migone F, Dai J, Schneider A, Serra D, Chen Y T, Amalfitano A
Abstract excerpt
Glycogen storage disease type II (GSD-II) is a lysosomal storage disorder in which the lack of human acid-alpha glucosidase (hGAA) activity results in massive accumulations of glycogen in cardiac and skeletal muscle fibers. Affected individuals die of cardiorespiratory failure secondary to the skeletal and/or cardiac muscle involvement. Recombinant hGAA enzyme replacement therapy (ERT) is currently in clinical...
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