Title : Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II.

Pub. Date : 2007 Jun

PMID : 17459751






4 Functional Relationships(s)
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1 The purpose of the studies presented here was to describe some of the preclinical development of idursulfase using the I2S knock-out mouse model of MPS II designed to study the effect of dose and various dosing regimens of idursulfase on urine and tissue GAG levels. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens
2 The purpose of the studies presented here was to describe some of the preclinical development of idursulfase using the I2S knock-out mouse model of MPS II designed to study the effect of dose and various dosing regimens of idursulfase on urine and tissue GAG levels. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens
3 Results showed that idursulfase, at several doses and at several dosing frequencies, caused a reduction in tissue and urine GAG levels in a dose-dependent manner. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens
4 These studies also demonstrated that after IV administration, idursulfase is biologically active in the IdS-KO mouse model and is transported to key target tissues, reaching the lysosomes in an active form, and degrading the accumulated GAG. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens