Search Results - "Terri M. Christianson"
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Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
Published in Proceedings of the National Academy of Sciences - PNAS (14-10-2014)“…Significance Mucopolysaccharidosis type IIIB (MPS IIIB) is a devastating and currently untreatable disease affecting mainly the brain. The cause is lack of the…”
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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B
Published in Drug delivery and translational research (01-04-2020)“…BMN 250 is being developed as enzyme replacement therapy for Sanfilippo type B, a primarily neurological rare disease, in which patients have deficient…”
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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
Published in Molecular therapy. Methods & clinical development (15-09-2017)“…Sanfilippo syndrome type B (mucopolysaccharidosis IIIB), caused by inherited deficiency of α- -acetylglucosaminidase (NAGLU), required for lysosomal…”
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Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice
Published in The Journal of biological chemistry (25-09-2020)“…Autosomal recessive mutations in the galactosidase β1 (GLB1) gene cause lysosomal β-gal deficiency, resulting in accumulation of galactose-containing…”
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Intermittent enzyme replacement therapy with recombinant human β-galactosidase prevents neuraminidase 1 deficiency
Published in The Journal of biological chemistry (25-09-2020)“…Mutations in the galactosidase β 1 (GLB1) gene cause lysosomal β-galactosidase (β-Gal) deficiency and clinical onset of the neurodegenerative lysosomal storage…”
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Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction
Published in The Journal of biological chemistry (01-12-2022)“…Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder caused by N-sulfoglucosamine sulfohydrolase (SGSH) deficiency. SGSH removes the…”
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Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency
Published in The Journal of biological chemistry (13-11-2020)Get full text
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Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B
Published in Molecular therapy. Methods & clinical development (13-09-2019)“…Sanfilippo syndrome type B, or mucopolysaccharidosis IIIB (MPS IIIB), is a rare autosomal recessive lysosomal storage disease caused by a deficiency of…”
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Overexpression of inactive arylsulphatase mutants and in vitro activation by light-dependent oxidation with vanadate
Published in Biochemical journal (01-09-2004)“…Arylsulphatases B (ASB) and A (ASA) are subject to a unique post-translational modification that is required for their function. The modification reaction,…”
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Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase
Published in The Journal of biological chemistry (13-08-2004)“…Enzyme replacement therapy for lysosomal storage disorders depends on efficient uptake of recombinant enzyme into the tissues of patients. This uptake is…”
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Lipoprotein Receptor Binding, Cellular Uptake, and Lysosomal Delivery of Fusions between the Receptor-associated Protein (RAP) and α-l-Iduronidase or Acid α-Glucosidase
Published in The Journal of biological chemistry (13-08-2004)“…Enzyme replacement therapy for lysosomal storage disorders depends on efficient uptake of recombinant enzyme into the tissues of patients. This uptake is…”
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