ArticleCommunications medicine2025
N-glycan-modified α-L-iduronidase produced by transgenic silkworms ameliorates clinical signs in a Japanese macaque with mucopolysaccharidosis I.
Article in Communications medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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The trial behind it
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Who cites it
3 citing papers in PubMed.
- Application of Transgenic Silk Fibroin to Biomaterials.Biotechnology and bioengineering · 2026Review
- AAV-based gene therapy with modified HEXB confers lasting therapeutic benefits in GM2 gangliosidosis models.Cell reports. Medicine · 2026Article
- Robust and Reproducible Monoclonal Antibody Production Using a Transgenic Silkworm System.International journal of molecular sciences · 2025Article
Corrections and comments
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Authors and funding
27 authors.
Funding
Abstract
backgroundMucopolysaccharidosis 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 N-glycans attached to therapeutic enzymes produced by mammalian cell lines has been clinically applied to several LSDs. Recent studies suggested an unidentified delivery pathway mediated by sialic acid-containing N-glycans. However, more economical platform development is required to produce large quantities of recombinant enzymes. Transgenic silkworms have been established as low-cost systems for expressing recombinant glycoproteins. Microbial endo-β-N-acetylglucosaminidases (ENGases) enable the transglycosylation of N-glycans to other types.
methodsWe purified recombinant human IDUA from IDUA transgenic silkworm cocoons and performed ENGase-mediated transglycosylation. Furthermore, we performed intravenous enzyme replacement therapy in a Japanese macaque MPS I non-human primate model carrying a homozygous IDUA missense mutation.
resultsHere we show the establishment of IDUA transgenic silkworms and purification of recombinant human IDUA from cocoons. As M6P- and sialic acid-containing N-glycans are not attached to purified hIDUA, we perform ENGase-mediated transglycosylation to obtain hIDUAs with M6P- and sialic acid-containing N-glycans (neoglyco-hIDUAs). Furthermore, we perform intravenous neoglyco-hIDUA replacement therapy in MPS I non-human primate model and succeed in improving the clinical signs and reducing the urinary glycosaminoglycan (GAG) levels.
conclusionsThese glycotechnologies using transgenic silkworms and ENGases are expected to serve as platforms for developing therapeutic glycoproteins.
Identifiers
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Registered trials
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