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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Shranjeno v:
Bibliografske podrobnosti
izdano v:J Inherit Metab Dis
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: Springer Netherlands 2015
Teme:
Original Article
Online dostop: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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