Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II
AR Garcia, JM DaCosta, J Pan, J Muenzer… - Molecular genetics and …, 2007 - Elsevier
Mucopolysaccharidosis II (MPS II; Hunter syndrome) is an X-linked metabolic disorder
caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S), which
catalyzes the catabolism of glycosaminoglycans (GAG) by cleaving the O-linked sulfate from
dermatan sulfate and heparan sulfate. Recently, enzyme replacement therapy (ERT) with
recombinant human I2S (Elaprase™(idursulfase), Shire Human Genetic Therapies, Inc.),
has been approved in the US and European Union for the treatment and management of …
caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S), which
catalyzes the catabolism of glycosaminoglycans (GAG) by cleaving the O-linked sulfate from
dermatan sulfate and heparan sulfate. Recently, enzyme replacement therapy (ERT) with
recombinant human I2S (Elaprase™(idursulfase), Shire Human Genetic Therapies, Inc.),
has been approved in the US and European Union for the treatment and management of …