Coexpression of formylglycine-generating enzyme is essential for synthesis and secretion of functional arylsulfatase A in a mouse model of metachromatic leukodystrophy.

@article{Takakusaki2005CoexpressionOF,
  title={Coexpression of formylglycine-generating enzyme is essential for synthesis and secretion of functional arylsulfatase A in a mouse model of metachromatic leukodystrophy.},
  author={Yukiko Takakusaki and Sanae Hisayasu and Yukihiko Hirai and Takashi L Shimada},
  journal={Human gene therapy},
  year={2005},
  volume={16 8},
  pages={929-36}
}
Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder involving inherited deficiency of arylsulfatase A (ASA). The disease is characterized by progressive demyelination and widespread deposition of sulfatide in both the central and peripheral nervous systems. Direct injection of viral vector through the blood-brain barrier is a possible gene therapy approach to MLD. However, to treat all brain cells, it is essential to secrete a sufficient amount of functional ASA from limited… CONTINUE READING

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