Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts

Enzyme replacement therapy for mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo B syndrome) has been hindered by inadequate mannose 6-phosphorylation and cellular uptake of recombinantly produced human α-N-acetyl-glucosamindase (rhNAGLU). We expressed and characterized a modified, recombinant h...

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Detalhes bibliográficos
Main Authors: Kan, Shih-hsin, Troitskaya, Larisa A., Sinow, Carolyn S., Haitz, Karyn, Todd, Amanda K., Di Stefano, Ariana, Le, Steven Q., Dickson, Patricia I., Tippin, Brigette L.
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3993987/
https://ncbi.nlm.nih.gov/pubmed/24266751
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1042/BJ20130845
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