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

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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Bibliographic Details
Main Authors: Mika Aoyagi-Scharber, Danielle Crippen-Harmon, Roger Lawrence, Jon Vincelette, Gouri Yogalingam, Heather Prill, Bryan K. Yip, Brian Baridon, Catherine Vitelli, Amanda Lee, Olivia Gorostiza, Evan G. Adintori, Wesley C. Minto, Jeremy L. Van Vleet, Bridget Yates, Sara Rigney, Terri M. Christianson, Pascale M.N. Tiger, Melanie J. Lo, John Holtzinger, Paul A. Fitzpatrick, Jonathan H. LeBowitz, Sherry Bullens, Brett E. Crawford, Stuart Bunting
Format: Article
Language:English
Published: Elsevier 2017-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
lysosomal storage disorder
mucopolysaccharidosis IIIB
MPS IIIB
Sanfilippo B
enzyme replacement therapy
intracerebroventricular delivery
glycosaminoglycan
neurodegeneration
mental retardation
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300748

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http://www.sciencedirect.com/science/article/pii/S2329050117300748

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