Search Results - "Yoden, Eiji"
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A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II
Published in Molecular therapy (02-05-2018)“…Mucopolysaccharidosis II (MPS II) is an X-linked recessive lysosomal storage disease caused by mutations in the iduronate-2-sulfatase (IDS) gene. Since IDS…”
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Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice
Published in Molecular therapy (05-05-2021)“…Mucopolysaccharidosis II (MPS II), a lysosomal storage disease caused by mutations in iduronate-2-sulfatase (IDS), is characterized by a wide variety of…”
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Nonclinical safety evaluation of pabinafusp alfa, an anti-human transferrin receptor antibody and iduronate-2-sulfatase fusion protein, for the treatment of neuronopathic mucopolysaccharidosis type II
Published in Molecular genetics and metabolism reports (01-06-2021)“…Pabinafusp alfa is a fusion protein comprising a humanized anti-human transferrin receptor (TfR) antibody and human iduronate-2-sulfatase. It was developed as…”
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Non-clinical evaluation of JR-051 as a biosimilar to agalsidase beta for the treatment of Fabry disease
Published in Molecular genetics and metabolism (01-09-2018)“…Fabry disease (FD) is an X-linked lysosomal storage disease. It is caused by deficiency of the enzyme α-galactosidase A (α-Gal A), which leads to excessive…”
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Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice
Published in Molecular therapy. Methods & clinical development (08-06-2023)“…Mucopolysaccharidosis I (MPS I), a lysosomal storage disease caused by dysfunction of α-L-iduronidase (IDUA), is characterized by the deposition of dermatan…”
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Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice
Published in Molecular pharmaceutics (06-11-2023)“…Mucopolysaccharidoses (MPSs) make up a group of lysosomal storage diseases characterized by the aberrant accumulation of glycosaminoglycans throughout the…”
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Recovery of retinal function in MPS II mice by treatment with pabinafusp alfa
Published in Molecular genetics and metabolism (01-02-2024)Get full text
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Life-span extension in Krabbe disease mice by treatment with a transferrin receptor-targeted galactocerebrosidase
Published in Molecular genetics and metabolism (01-02-2023)Get full text
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The characterization of mouse model of mucopolysaccharidosis type II
Published in Molecular genetics and metabolism (01-02-2015)Get full text
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Novel quantification methods for globotriaosylceramide and globotriaosylsphingosine as biomarkers of Fabry disease
Published in Molecular genetics and metabolism (01-02-2015)Get full text
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