Characterization and pharmacokinetic study of recombinant human N-acetylgalactosamine-6-sulfate sulfatase
S Tomatsu, AM Montaño, M Gutierrez, JH Grubb… - Molecular genetics and …, 2007 - Elsevier
Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder caused by a
deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). The aims of this study
were to establish Chinese hamster ovary (CHO) cells overexpressing recombinant human
GALNS (rhGALNS) and to assess pharmacokinetics and tissue distribution of purified
enzymes by using MPS IVA knock-out mouse (Galns−/−). The CHO-cell derived rhGALNS
was purified from the media by a two-step affinity chromatography procedure. The rhGALNS …
deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). The aims of this study
were to establish Chinese hamster ovary (CHO) cells overexpressing recombinant human
GALNS (rhGALNS) and to assess pharmacokinetics and tissue distribution of purified
enzymes by using MPS IVA knock-out mouse (Galns−/−). The CHO-cell derived rhGALNS
was purified from the media by a two-step affinity chromatography procedure. The rhGALNS …