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Development of Substrate Degradation Enzyme Therapy for Mucopolysaccharidosis IVA Murine Model
Mucopolysaccharidosis IVA (MPS IVA) is caused by a deficiency of the lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Conventional enzyme replacement therapy (ERT) is approved for MPS IVA. However, the fact that the infused enzyme cannot penetrate avascular lesions in cartilage le...
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| Publicado no: | Int J Mol Sci |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6747109/ https://ncbi.nlm.nih.gov/pubmed/31450640 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20174139 |
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