Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.

@article{Huynh2012BiochemicalEF,
  title={Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.},
  author={Ha T. Huynh and Jeffrey H. Grubb and Carole Vogler and William S. Sly},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={2012},
  volume={109 42},
  pages={17022-7}
}
Enzyme replacement therapy has been used successfully in many lysosomal storage diseases. However, correction of brain storage has been limited by the inability of infused enzyme to cross the blood-brain barrier (BBB). We recently reported that PerT-GUS, a form of β-glucuronidase (GUS) chemically modified to eliminate its uptake and clearance by carbohydrate-dependent receptors, crossed the BBB and cleared neuronal storage in an immunotolerant model of murine mucopolysaccharidosis (MPS) type… CONTINUE READING
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