Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

@article{Watson1998TreatmentOL,
  title={Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus},
  author={G N A Watson and J A Sayles and C Chen and S Elliger and C Elliger and N. L. Raju and G Kurtzman and G Podsakoff},
  journal={Gene Therapy},
  year={1998},
  volume={5},
  pages={1642-1649}
}
Mucopolysaccharidosis type VII (MPS VII) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase (GUS). We used a recombinant adeno-associated virus vector (AAV-GUS) to deliver GUS cDNA to MPS VII mice. The route of vector administration had a dramatic effect on the extent and distribution of GUS activity. Intramuscular injection of AAV-GUS resulted in high, localized production of GUS, while intravenous administration produced low GUS activity in several tissues. This… CONTINUE READING

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Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
Mucopolysaccharidosis type VII ( MPS VII ) is a lysosomal storage disease caused by a genetic deficiency of β-glucuronidase ( GUS ) .
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