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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Publicat a:Mol Ther Methods Clin Dev
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5480280/
https://ncbi.nlm.nih.gov/pubmed/28664165
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.05.009
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