Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells.

@article{Marchal1993DisappearanceOL,
  title={Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells.},
  author={Vincent Mar{\'e}chal and Nadia Naffakh and Olivier Danos and Jean Michel Heard},
  journal={Blood},
  year={1993},
  volume={82 4},
  pages={1358-65}
}
Mice homozygous for the gusmps allele lack beta-glucuronidase activity and provide a useful model for human Mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. Bone marrow (BM) transplantation was shown to correct the metabolic defect and to increase the life span of diseased animals. We have used this murine model in a preclinical study aimed at evaluating whether the techniques currently available for gene transfer into large mammalian and human BM cells will provide… CONTINUE READING

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