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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Detalles Bibliográficos
Autores principales: Sawamoto, Kazuki, Tomatsu, Shunji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6747109/
https://www.ncbi.nlm.nih.gov/pubmed/31450640
http://dx.doi.org/10.3390/ijms20174139

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6747109/
https://www.ncbi.nlm.nih.gov/pubmed/31450640
http://dx.doi.org/10.3390/ijms20174139

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