Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice.

@article{Stein1999SystemicAC,
  title={Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice.},
  author={Colleen S. Stein and Abdi Ghodsi and Todd A Derksen and Beverly L. Davidson},
  journal={Journal of virology},
  year={1999},
  volume={73 4},
  pages={3424-9}
}
Mucopolysaccharidosis (MPS) type VII patients lack functional beta-glucuronidase, leading to systemic and central nervous system dysfunction. In this study we tested whether recombinant adenovirus that encodes beta-glucuronidase (Adbetagluc), delivered intravenously and into the brain parenchyma of MPS type VII mice, could provide long-term transgene expression and correction of lysosomal distension. We also tested whether systemic treatment with the immunosuppressive anti-CD40 ligand antibody… CONTINUE READING

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A. Ghodsi, G. Yang, +3 authors B. L. Davidson
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