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Article

Enzyme replacement therapy in a murine model of Morquio A syndrome

27 Nov 2007

Abstract excerpt

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....

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PMID: 18056156
DOI: 10.1093/hmg/ddm353

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