BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis

Sanfilippo syndrome type B (Sanfilippo B; Mucopolysaccharidosis type IIIB) occurs due to genetic deficiency of lysosomal alpha-N-acetylglucosaminidase (NAGLU) and subsequent lysosomal accumulation of heparan sulfate (HS), which coincides with devastating neurodegenerative disease. Because NAGLU expr...

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Detalles Bibliográficos
Autores principales: Yogalingam, Gouri, Luu, Amanda R., Prill, Heather, Lo, Melanie J., Yip, Bryan, Holtzinger, John, Christianson, Terri, Aoyagi-Scharber, Mika, Lawrence, Roger, Crawford, Brett E., LeBowitz, Jonathan H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6338363/
https://www.ncbi.nlm.nih.gov/pubmed/30657762
http://dx.doi.org/10.1371/journal.pone.0207836

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