Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice

Enzyme replacement therapy (ERT) is an effective treatment for several lysosomal storage disorders (LSDs). Intravenously infused enzymes are taken up by tissues through either the mannose 6-phosphate receptor (M6PR) or the mannose receptor (MR). It is generally believed that M6PR-mediated endocytosi...

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Vydáno v:J Inherit Metab Dis
Hlavní autoři: Shen, Jin-Song, Busch, Andreas, Day, Taniqua S., Meng, Xing-Li, Yu, Chun I., Dabrowska-Schlepp, Paulina, Fode, Benjamin, Niederkrüger, Holger, Forni, Sabrina, Chen, Shuyuan, Schiffmann, Raphael, Frischmuth, Thomas, Schaaf, Andreas
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Netherlands 2015
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4754329/
https://ncbi.nlm.nih.gov/pubmed/26310963
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-015-9886-9
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