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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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Foilsithe in:Int J Mol Sci
Main Authors: Sawamoto, Kazuki, Tomatsu, Shunji
Formáid: Artigo
Teanga:Inglês
Foilsithe: MDPI 2019
Ábhair:
Rochtain Ar Líne: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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