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Human Molecular Genetics, Vol. 7, Iss. 9, 1998-09 ,pp. : 1347-1354

Oxford University Press

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Enzyme therapy for lysosomal acid lipase deficiency in the mouse

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Human Molecular Genetics, Vol. 10, Iss. 16, 2001-08 ,pp. : 1639-1648

Oxford University Press

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-Mannosidosis mice: a model for the human lysosomal storage disease

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Adenovirus-mediated transfer of the acid α-glucosidase gene into fibroblasts, myoblasts and myotubes from patients with glycogen storage disease type II leads to high level expression of enzyme and corrects glycogen accumulation

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A Model of mRNA Splicing in Adult Lysosomal Storage Disease (Glycogenosis Type II)

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