Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome-Reference-Cited by-同舟云学术

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Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome

Published:2021-06 Issue:2 Volume:133 Page:185-192
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Kan Shih-hsin,Elsharkawi Ibrahim,Le Steven Q.,Prill Heather,Mangini Linley,Cooper Jonathan D.,Lawrence Roger,Sands Mark S.,Crawford Brett E.,Dickson Patricia I.

Funder

National Institutes of Health

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference35 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Sanfilippo syndrome: a mini-review;Valstar;J. Inherit. Metab. Dis.,2008

3. Sanfilippo syndrome: causes, consequences, and treatments;Fedele;Appl. Clin. Genet.,2015

4. The molecular basis of Sanfilippo syndrome type B;Zhao;Proc. Natl. Acad. Sci. U. S. A.,1996

5. Sanfilippo type B syndrome (mucopolysaccharidosis III B): allelic heterogeneity corresponds to the wide spectrum of clinical phenotypes;Weber;Eur. J. Hum. Genet.,1999

Cited by 2 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Mucopolysaccharidoses and the blood–brain barrier;Fluids and Barriers of the CNS;2022-09-19

2. Recombinant NAGLU-IGF2 prevents physical and neurological disease and improves survival in Sanfilippo B syndrome;2021-08-08

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