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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Netherlands 2015
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Original Article
Acesso em linha: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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