Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: a potential therapeutic approach for Sanfilippo disease

@article{Dziedzic2010ImpairmentOG,
  title={Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: a potential therapeutic approach for Sanfilippo disease},
  author={Dariusz Dziedzic and Grzegorz Węgrzyn and Joanna Jak{\'o}bkiewicz-Banecka},
  journal={European Journal of Human Genetics},
  year={2010},
  volume={18},
  pages={200-205}
}
Mucopolysaccharidoses (MPS) are severe inherited metabolic disorders from the group of lysosomal storage diseases. They are caused by deficiency in the activity of enzymes involved in the degradation of glycosaminoglycans (GAGs) and resultant accumulation of these compounds in the cells of patients. Although enzyme replacement therapy has become available for some MPS types (MPS I, MPS II and MPS VI), this treatment is not efficient when neurological symptoms occur, especially in MPS III… CONTINUE READING