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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Détails bibliographiques
Publié dans:J Inherit Metab Dis
Auteurs principaux: 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
Langue:Inglês
Publié: Springer Netherlands 2015
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Original Article
Accès en ligne: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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