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

Mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) is caused by a deficiency in α-N-acetylglucosaminidase (NAGLU) activity, which leads to the accumulation of heparan sulfate (HS). MPS IIIB causes progressive neurological decline, with affected patients having an expected lifespan of...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2021
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8195848/
https://ncbi.nlm.nih.gov/pubmed/33839004
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2021.03.013
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