Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B

Sanfilippo syndrome type B, or mucopolysaccharidosis IIIB (MPS IIIB), is a rare autosomal recessive lysosomal storage disease caused by a deficiency of α-N-acetylglucosaminidase (NAGLU). Deficiency in NAGLU disrupts the lysosomal turnover of heparan sulfate (HS), which results in the abnormal accumu...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Prill, Heather, Luu, Amanda, Yip, Bryan, Holtzinger, John, Lo, Melanie J., Christianson, Terri M., Yogalingam, Gouri, Aoyagi-Scharber, Mika, LeBowitz, Jonathan H., Crawford, Brett E., Lawrence, Roger
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6606967/
https://ncbi.nlm.nih.gov/pubmed/31309128
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.05.008
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