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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Sanfilippo syndrome type B (mucopolysaccharidosis IIIB), caused by inherited deficiency of α-N-acetylglucosaminidase (NAGLU), required for lysosomal degradation of heparan sulfate (HS), is a pediatric neurodegenerative disorder with no approved treatment. Intracerebroventricular (ICV) delivery of a...

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Detalles Bibliográficos
Autores principales:	Aoyagi-Scharber, Mika, Crippen-Harmon, Danielle, Lawrence, Roger, Vincelette, Jon, Yogalingam, Gouri, Prill, Heather, Yip, Bryan K., Baridon, Brian, Vitelli, Catherine, Lee, Amanda, Gorostiza, Olivia, Adintori, Evan G., Minto, Wesley C., Van Vleet, Jeremy L., Yates, Bridget, Rigney, Sara, Christianson, Terri M., Tiger, Pascale M.N., Lo, Melanie J., Holtzinger, John, Fitzpatrick, Paul A., LeBowitz, Jonathan H., Bullens, Sherry, Crawford, Brett E., Bunting, Stuart
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5480280/ https://www.ncbi.nlm.nih.gov/pubmed/28664165 http://dx.doi.org/10.1016/j.omtm.2017.05.009

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