{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,9]],"date-time":"2026-05-09T04:30:10Z","timestamp":1778301010482,"version":"3.51.4"},"reference-count":49,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2011,3,17]],"date-time":"2011-03-17T00:00:00Z","timestamp":1300320000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2011,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Lysosomal storage diseases (LSDs) are metabolic disorders caused by enzyme deficiencies that lead to lysosomal accumulation of undegraded substrates. Enzyme replacement therapies (ERT) have been developed as treatments for patients with Gaucher, Niemann\u2010Pick, Fabry, and Pompe diseases. Depending on the disease, the corresponding therapeutic enzyme is designed to be internalized by diseased cells through receptor\u2010mediated endocytosis via macrophage mannose receptors (MMR) or mannose\u20106\u2010phosphate receptors (M6PR). Enzymes developed to treat Gaucher and Niemann\u2010Pick diseases are meant to target MMR\u2010expressing cells, and in the case of Cerezyme [recombinant human \u03b2\u2010glucocerebrosidase (rh\u03b2GC)] for treating Gaucher disease, glycans on the enzyme are modified to increase specificity toward this receptor. Due to heterogeneity in glycosylation on enzymes intended to target the M6PR, however, there may also be some unintended targeting to MMR\u2010expressing cells, which could act as unwanted sinks. Examples include Fabrazyme [recombinant human \u03b1\u2010galactosidase A (rh\u03b1Gal)] for treating Fabry disease and Myozyme [recombinant human acid \u03b1\u2010glucosidase (rhGAA)] for treating Pompe disease. It is therefore of great interest to better understand the cell type and tissue distribution of MMR in murine LSD models used to evaluate ERT efficacy and mechanism of action. In this study, we generated affinity\u2010purified polyclonal antibody against murine MMR and used it to carry out a systematic examination of MMR protein localization in murine models of Gaucher, Niemann\u2010Pick, Fabry, and Pompe diseases. Using immunohistochemistry, immunofluorescence, and confocal microscopy, we examined MMR distribution in liver, spleen, lung, kidney, heart, diaphragm, quadriceps, and triceps in these animal models and compared them with MMR distribution in wild\u2010type mice.<\/jats:p>","DOI":"10.1007\/s10545-011-9285-9","type":"journal-article","created":{"date-parts":[[2011,3,16]],"date-time":"2011-03-16T17:33:54Z","timestamp":1300296834000},"page":"795-809","source":"Crossref","is-referenced-by-count":31,"title":["Biotherapeutic target or sink: analysis of the macrophage mannose receptor tissue distribution in murine models of lysosomal storage diseases"],"prefix":"10.1002","volume":"34","author":[{"given":"Xin Sheen","family":"Zhang","sequence":"first","affiliation":[{"name":"Department of Pathology, Genzyme Corporation 5 The Mountain Road Framingham MA 01701 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