Associated Papers
Engineering stable and non-immunogenic immunoenzymes for cancer therapy via in situ generated prodrugs.
Tseng YH et al.
Journal of Controlled Release : Official Journal of the Controlled Release Society, 2024
Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII.
Cadaoas J et al.
Molecular Genetics and Metabolism, 2020
Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome).
Tomatsu S et al.
Human Mutation, 2009
Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice.
Montaño AM et al.
Molecular Genetics and Metabolism, 2008
Development of MPS IVA mouse (Galnstm(hC79S.mC76S)slu) tolerant to human N-acetylgalactosamine-6-sulfate sulfatase.
Tomatsu S et al.
Human Molecular Genetics, 2005
Missense models [Gustm(E536A)Sly, Gustm(E536Q)Sly, and Gustm(L175F)Sly] of murine mucopolysaccharidosis type VII produced by targeted mutagenesis.
Tomatsu S et al.
Proceedings of the National Academy of Sciences of the United States of America, 2002
[The active site of human glucocerebrosidase: structural predictions and experimental validations].
Fabrega S et al.
Journal De La Societe De Biologie, 2002
Gene therapy for lysosomal storage disorders.
Yew NS et al.
Current Opinion in Molecular Therapeutics, 2001
Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.
Sly WS et al.
Proceedings of the National Academy of Sciences of the United States of America, 2001
Active site residues of human beta-glucuronidase. Evidence for Glu(540) as the nucleophile and Glu(451) as the acid-base residue.
Islam MR et al.
The Journal of Biological Chemistry, 1999
Prospects for enzyme replacement for lysosomal storage diseases.
Sly WS
Birth Defects Original Article Series, 1981
Select a paper to view details