Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery

@article{Richard2009WidespreadBC,
  title={Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery},
  author={Magali Richard and Audrey Arfi and J{\'e}r{\'e}mie Seguin and Christelle Gandolphe and Daniel Scherman},
  journal={Gene Therapy},
  year={2009},
  volume={16},
  pages={746-756}
}
Mucopolysaccharidosis type VII (MPS VII) is a lysosomal storage disease caused by a deficiency of the acid hydrolase β-glucuronidase. MPS VII mice develop progressive lysosomal accumulation of glycosaminoglycans (GAGs) within multiple organs, including the brain. Using this animal model, we compared two plasmid gene administration techniques: muscle electrotransfer and liver-directed transfer using hydrodynamic injection. We have evaluated both the expression kinetics and the biodistribution of… CONTINUE READING

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