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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Detalhes bibliográficos

Principais autores:	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
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2017-09-01
coleção:	Molecular Therapy: Methods & Clinical Development
Assuntos:	lysosomal storage disorder mucopolysaccharidosis IIIB MPS IIIB Sanfilippo B enzyme replacement therapy intracerebroventricular delivery glycosaminoglycan neurodegeneration mental retardation
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2329050117300748
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