Article

N-glycan-modified α-L-iduronidase produced by transgenic silkworms ameliorates symptoms in a Japanese macaque with mucopolysaccharidosis I

2024-06-05

Abstract excerpt

<title>Abstract</title> <p>Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disorder (LSD) caused by recessive mutations in the α-L-iduronidase (IDUA) gene. Enzyme replacement therapy (ERT) utilizing terminal mannose 6-phosphate (M6P)-carrying <italic>N</italic>-glycans attached to therapeutic enzymes produced by mammalian cell lines has been clinically applied to several LSDs. Recent studie...

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Literature Corpus work: 8d52cc82-3d86-5c1b-9d62-1dd9f4ae8f87
DOI: 10.21203/rs.3.rs-4419175/v1

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