Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

@article{Hennig2003IntravitrealGT,
  title={Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.},
  author={Anne K. Hennig and Beth Levy and Judith Mosinger Ogilvie and Carole A. Vogler and Nancy J. Galvin and Steven Bassnett and Mark S Sands},
  journal={The Journal of neuroscience : the official journal of the Society for Neuroscience},
  year={2003},
  volume={23 8},
  pages={3302-7}
}
The mucopolysaccharidoses (MPSs) are lysosomal storage diseases resulting from impaired catabolism of sulfated glycosaminoglycans. MPS VII mice lack lysosomal beta-glucuronidase (GUSB) activity, leading to the accumulation of partially degraded chondroitin, dermatan, and heparan sulfates in most tissues. Consequently, these mice develop most of the symptoms exhibited by human MPS VII patients, including progressive visual and cognitive deficits. To investigate the effects of reducing lysosomal… CONTINUE READING

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