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Single-cell RNA sequencing reveals important role of monocytes and macrophages during mucopolysaccharidosis treatment. [PDF]

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Zhu G, Xie Y, Li L, Li R, Sun Y, Zhou T, Cun Y.   +6 more
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Identification of iduronate-2-sulfatase in mouse pancreatic islets

American Journal of Physiology-Endocrinology and Metabolism, 2004
The lysosomal enzyme iduronate-2-sulfatase (IDS) is expressed in pancreatic islets and is responsible for degradation of proteoglycans, such as perlecan and dermatan sulfate. To determine the role of IDS in islets, expression and regulation of the gene and localization of the enzyme were investigated in mouse pancreatic islets and clonal cells. The Ids
I, Coronado-Pons, A, Novials, S, Casas, A, Clark, R, Gomis   +4 more
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“Supercharged Cells” for Delivery of Recombinant Human Iduronate-2-Sulfatase

Molecular Genetics and Metabolism, 2000
Expression of iduronate-2-sulfatase (IDS) from three different promoters in four retroviral vectors was studied in peripheral blood lymphocytes from patients with Hunter syndrome (PBL(MPS)), i.e., the LTR in vectors L2SN and L2, avian beta-actin promoter in LB2, and the CMV early promoter in LNC2.
D, Pan, J J, Jonsson, S E, Braun, R S, McIvor, C B, Whitley   +4 more
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Brain-Penetrating IgG-Iduronate 2-Sulfatase Fusion Protein for the Mouse

Drug Metabolism and Disposition, 2012
Mucopolysaccharidosis (MPS) type II (Hunter's syndrome) is caused by mutations in the iduronate 2-sulfatase (IDS) fusion protein. MPS-II affects the brain, and enzyme replacement therapy is not effective in the brain, because the enzyme does not cross the blood-brain barrier.
Qing-Hui, Zhou, Ruben J, Boado, Jeff Zhiqiang, Lu, Eric Ka-Wai, Hui, William M, Pardridge   +4 more
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Intrathecal delivery of recombinant human iduronate-2-sulfatase (idursulfase-IT)

Molecular Genetics and Metabolism, 2011
Background: Idursulfase has limited passage through the bloodbrain barrier when administered intravenously. Objectives: We have undertaken a program to develop an intrathecal (IT) formulation of idursulfase to target CNS manifestations of MPS II. Methods: In a safety study, single bolus injections of idursulfase-IT 3 mg (n=12), 30 mg (n=6), or 100 mg ...
P. Calias, J. Pan, Y. Huang, M. Alessandrini, N. Liu, R.W. Pfeifer, T.L. Wright, B.R. Felice, J. Powell, N. Savioli, J. Lotterhand, M.W. Heartlein   +11 more
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A novel functional role of iduronate-2-sulfatase in zebrafish early development

Matrix Biology, 2010
Sulfated glycosaminoglycan chains of extracellular matrix and cell membrane-tethered proteoglycans exert specific cellular functions by interacting with a broad spectrum of morphogens and growth factors. In humans, a congenital impaired catabolism of sulfated glycosaminoglycans is associated with severe metabolic disorders.
MORO, ENRICO, TOMANIN R, FRISO A, MODENA N, TISO, NATASCIA, SCARPA, MAURIZIO, ARGENTON, FRANCESCO   +6 more
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Genetic Engineering of a Bifunctional IgG Fusion Protein with Iduronate-2-Sulfatase

Bioconjugate Chemistry, 2009
Iduronate-2-sulfatase (IDS) is a lysosomal sulfatase that prevents the accumulation within the brain of glycosoaminoglycans. However, IDS does not cross the blood-brain barrier (BBB). To enable BBB transport, human IDS, minus its signal peptide, was fused to the carboxyl terminus of the heavy chain of a chimeric monoclonal antibody (mAb) to the human ...
Jeff Zhiqiang, Lu, Eric Ka-Wai, Hui, Ruben J, Boado, William M, Pardridge   +3 more
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DNA deletion confined to the iduronate-2-sulfatase promoter abolishes IDS gene expression

Human Mutation, 1998
Deficiency of the enzyme iduronate-2-sulfatase (IDS) results in Hunter syndrome, an X-linked recessive lysosomal storage disorder. In this study, analysis of a patient with features of moderate to severe Hunter syndrome identified a 178-bp deletion upstream of IDS exon 1 spanning a predicted promoter element.
K M, Timms, L E, Huckett, J W, Belmont, S K, Shapira, R A, Gibbs   +4 more
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