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It is generally believed that M6PR\u2010mediated endocytosis is a key mechanism for ERT in treating LSDs that affect the non\u2010macrophage cells of visceral organs. However, the therapeutic efficacy of MR\u2010mediated delivery of mannose\u2010terminated enzymes in these diseases has not been fully evaluated. We tested the effectiveness of a non\u2010phosphorylated \u03b1\u2010galactosidase A produced from moss (referred to as moss\u2010aGal) in vitro and in a mouse model of Fabry disease. Endocytosis of moss\u2010aGal was MR\u2010dependent. Compared to agalsidase alfa, a phosphorylated form of \u03b1\u2010galactosidase A, moss\u2010aGal was more preferentially targeted to the kidney. Cellular localization of moss\u2010aGal and agalsidase alfa in the heart and kidney was essentially identical. A single injection of moss\u2010aGal led to clearance of accumulated substrate in the heart and kidney to an extent comparable to that achieved by agalsidase alfa. This study suggested that mannose\u2010terminated enzymes may be sufficiently effective for some LSDs in which non\u2010macrophage cells are affected, and that M6P residues may not always be a prerequisite for ERT as previously considered.<\/jats:p>","DOI":"10.1007\/s10545-015-9886-9","type":"journal-article","created":{"date-parts":[[2015,8,26]],"date-time":"2015-08-26T07:29:33Z","timestamp":1440574173000},"page":"293-303","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":98,"title":["Mannose receptor\u2010mediated delivery of moss\u2010made \u03b1\u2010galactosidase A efficiently corrects enzyme deficiency in Fabry mice"],"prefix":"10.1002","volume":"39","author":[{"given":"Jin\u2010Song","family":"Shen","sequence":"first","affiliation":[{"name":"Institute of Metabolic Disease Baylor Research Institute 3812 Elm Street Dallas TX 75226 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