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

26 Aug 2015

Abstract excerpt

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 endocytosis is a key mechanism for ERT in treating LSDs that affect the non-macrophage cells of visceral organs. However, the...

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PMID: 26310963
DOI: 10.1007/s10545-015-9886-9

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