Enzyme replacement therapy in a murine model of Morquio A syndrome.

  title={Enzyme replacement therapy in a murine model of Morquio A syndrome.},
  author={Shunji Tomatsu and Adriana Maria Monta{\~n}o and Amiko Ohashi and M{\'o}nica A Guti{\'e}rrez and Hirotaka Oikawa and Toshihiro Oguma and Vũ Ch{\'i} Dũng and Tatsuo Nishioka and Tadao Orii and William S. Sly},
  journal={Human molecular genetics},
  volume={17 6},
Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), leading to accumulation of keratan sulfate (KS) and chrondroitin-6-sulfate. The pharmacokinetics and biodistributions were determined for two recombinant human GALNSs produced in CHO cell lines: native GALNS and sulfatase-modifier-factor 1 (SUMF1) modified GALNS. Preclinical studies of enzyme replacement therapy (ERT) by using two GALNS enzymes were… CONTINUE READING