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Enzyme replacement therapy (ERT) is one of the most significant advancements and breakthroughs in treating FD. However, limited resources and the high cost of ERT might prevent patients from receiving prompt and effective therapy, thereby resulting in severe complications. Future progress in ERT can uncover promising treatment options. In this study, we developed and validated a recombinant enzyme (Lanzyme) based on a CHO-S cell system to provide a new potential option for FD therapy. Our results indicated that Lanzyme was heavily glycosylated, and its highest activity was similar to a commercial enzyme (Fabrazyme\u00ae). Our pharmacokinetic assessment revealed that the half-life of Lanzyme was up to 11 min, which is nearly twice that of the commercial enzyme. In vivo experiments revealed that Lanzyme treatment sharply decreased the accumulation levels of Gb3 and lyso-Gb3 in various tissues of FD model mice, with superior or comparable therapeutic effects to Fabrazyme\u00ae. Based on these data, Lanzyme may represent a new and promising treatment approach for FD. Building this enzyme production system for ERT can offer additional choice, potentially with enhanced efficacy, for the benefit of patients with FD.<\/jats:p>","DOI":"10.3390\/biom13010053","type":"journal-article","created":{"date-parts":[[2022,12,28]],"date-time":"2022-12-28T08:38:19Z","timestamp":1672216699000},"page":"53","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Development of Lanzyme as the Potential Enzyme Replacement Therapy Drug for Fabry Disease"],"prefix":"10.3390","volume":"13","author":[{"given":"Mulan","family":"Deng","sequence":"first","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongyu","family":"Zhou","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhicheng","family":"Liang","sequence":"additional","affiliation":[{"name":"The School of Medicine, South China University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhaoyang","family":"Li","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanping","family":"Wang","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wanyi","family":"Guo","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"April Yuanyi","family":"Zhao","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanghong","family":"Li","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yunping","family":"Mu","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allan Zijian","family":"Zhao","sequence":"additional","affiliation":[{"name":"The School of Biomedical and Pharmaceutical Sciences, Guangdong University of Technology, Guangzhou 510000, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,12,27]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Dardis, A., and Buratti, E. (2018). Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders. Genes, 9.","DOI":"10.3390\/genes9020073"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"114464","DOI":"10.1016\/j.addr.2022.114464","article-title":"Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders","volume":"188","author":"Parlanti","year":"2022","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"112","DOI":"10.15252\/emmm.201606965","article-title":"Lysosomal dysfunction disrupts presynaptic maintenance and restoration of presynaptic function prevents neurodegeneration in lysosomal storage diseases","volume":"9","author":"Sambri","year":"2017","journal-title":"EMBO Mol. Med."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1038\/s41572-018-0025-4","article-title":"Lysosomal storage diseases","volume":"4","author":"Platt","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"11","DOI":"10.3892\/ijmm.2012.1187","article-title":"New strategies for the treatment of lysosomal storage diseases (review)","volume":"31","author":"Parenti","year":"2013","journal-title":"Int. J. Mol. Med."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1007\/s40265-021-01486-1","article-title":"Fabry Disease: The Current Treatment Landscape","volume":"81","author":"Lenders","year":"2021","journal-title":"Drugs"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1056\/NEJM196705252762101","article-title":"Enzymatic defect in Fabry\u2019s disease. Ceramidetrihexosidase deficiency","volume":"276","author":"Brady","year":"1967","journal-title":"N. Engl. J. Med."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"1169","DOI":"10.1038\/mt.2015.87","article-title":"Coformulation of a Novel Human \u03b1-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice","volume":"23","author":"Xu","year":"2015","journal-title":"Mol. Ther."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"100454","DOI":"10.1016\/j.ymgmr.2019.100454","article-title":"The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts","volume":"19","author":"Germain","year":"2019","journal-title":"Mol. Genet. Metab. Rep."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1086\/318809","article-title":"A phase 1\/2 clinical trial of enzyme replacement in fabry disease: Pharmacokinetic, substrate clearance, and safety studies","volume":"68","author":"Eng","year":"2001","journal-title":"Am. J. Hum. Genet."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"2812","DOI":"10.1073\/pnas.0712309105","article-title":"Elevated globotriaosylsphingosine is a hallmark of Fabry disease","volume":"105","author":"Aerts","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1016\/j.ymgme.2010.03.020","article-title":"Plasma globotriaosylsphingosine as a biomarker of Fabry disease","volume":"100","author":"Togawa","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"741","DOI":"10.1016\/j.bbadis.2010.05.003","article-title":"Plasma globotriaosylsphingosine: Diagnostic value and relation to clinical manifestations of Fabry disease","volume":"1802","author":"Rombach","year":"2010","journal-title":"Biochim. Biophys. Acta"},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"716","DOI":"10.1016\/j.bbrc.2010.08.006","article-title":"Tissue and plasma globotriaosylsphingosine could be a biomarker for assessing enzyme replacement therapy for Fabry disease","volume":"399","author":"Togawa","year":"2010","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1186\/s13052-018-0562-1","article-title":"Enzyme replacement therapy: Efficacy and limitations","volume":"44","author":"Concolino","year":"2018","journal-title":"Ital. J. Pediatr."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"1464","DOI":"10.1056\/NEJM199105233242104","article-title":"Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher\u2019s disease","volume":"324","author":"Barton","year":"1991","journal-title":"N. Engl. J. Med."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"1913","DOI":"10.1073\/pnas.87.5.1913","article-title":"Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease","volume":"87","author":"Barton","year":"1990","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"13","DOI":"10.20517\/rdodj.2022.09","article-title":"Enzyme replacement therapy: Current challenges and drug delivery prospects via extracellular vesicles","volume":"1","author":"Silva","year":"2022","journal-title":"Rare Dis. Orphan Drugs J."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1038\/mt.2009.220","article-title":"The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease","volume":"18","author":"Khanna","year":"2010","journal-title":"Mol. Ther."},{"key":"ref_20","doi-asserted-by":"crossref","unstructured":"Sueoka, H., Aoki, M., Tsukimura, T., Togawa, T., and Sakuraba, H. (2015). Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an \u03b1-Galactosidase A Knockout Mouse, a Model of Fabry Disease. PLoS ONE, 10.","DOI":"10.1371\/journal.pone.0144958"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1007\/s00441-014-1922-9","article-title":"Depletion of globosides and isoglobosides fully reverts the morphologic phenotype of Fabry disease","volume":"358","author":"Porubsky","year":"2014","journal-title":"Cell Tissue Res."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1016\/0005-2744(77)90096-1","article-title":"alpha-galactosidase A from human placenta. Stability and subunit size","volume":"484","author":"Mayes","year":"1977","journal-title":"Biochim. Biophys. Acta"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"9994","DOI":"10.1016\/S0021-9258(19)86663-2","article-title":"Studies on human liver alpha-galactosidases. I. Purification of alpha-galactosidase A and its enzymatic properties with glycolipid and oligosaccharide substrates","volume":"254","author":"Dean","year":"1979","journal-title":"J. Biol. Chem."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"1307","DOI":"10.1016\/S0021-9258(19)69965-5","article-title":"Affinity purification of alpha-galactosidase A from human spleen, placenta, and plasma with elimination of pyrogen contamination. Properties of the purified splenic enzyme compared to other forms","volume":"256","author":"Bishop","year":"1981","journal-title":"J. Biol. Chem."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"908","DOI":"10.1002\/jimd.12228","article-title":"Developments in the treatment of Fabry disease","volume":"43","author":"Hollak","year":"2020","journal-title":"J. Inherit. Metab. Dis."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"159","DOI":"10.1016\/0378-1119(87)90119-3","article-title":"Expression of the human alpha-galactosidase A in Escherichia coli K-12","volume":"57","author":"Hantzopoulos","year":"1987","journal-title":"Gene"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1016\/0378-1119(94)90378-6","article-title":"Characterization of glycosylated and catalytically active recombinant human alpha-galactosidase A using a baculovirus vector","volume":"144","author":"Coppola","year":"1994","journal-title":"Gene"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1093\/glycob\/cwf096","article-title":"Production in yeast of alpha-galactosidase A, a lysosomal enzyme applicable to enzyme replacement therapy for Fabry disease","volume":"12","author":"Chiba","year":"2002","journal-title":"Glycobiology"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1007\/s10545-015-9886-9","article-title":"Mannose receptor-mediated delivery of moss-made \u03b1-galactosidase A efficiently corrects enzyme deficiency in Fabry mice","volume":"39","author":"Shen","year":"2016","journal-title":"J. Inherit. Metab. Dis."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/j.ymgme.2014.08.002","article-title":"Characterization of a chemically modified plant cell culture expressed human \u03b1-Galactosidase-A enzyme for treatment of Fabry disease","volume":"114","author":"Kizhner","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1007\/978-1-4939-6972-2_10","article-title":"The \u2018Omics Revolution in CHO Biology: Roadmap to Improved CHO Productivity","volume":"1603","author":"Dahodwala","year":"2017","journal-title":"Methods Mol. Biol."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1186\/1475-2859-8-17","article-title":"Microbial factories for recombinant pharmaceuticals","volume":"8","author":"Corchero","year":"2009","journal-title":"Microb. Cell Fact."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"1206","DOI":"10.1002\/btpr.637","article-title":"Integrated approach to produce a recombinant, His-tagged human \u03b1-galactosidase A in mammalian cells","volume":"27","author":"Corchero","year":"2011","journal-title":"Biotechnol. Prog."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1093\/glycob\/cwaa041","article-title":"How glycosylation affects glycosylation: The role of N-glycans in glycosyltransferase activity","volume":"30","author":"Mikolajczyk","year":"2020","journal-title":"Glycobiology"},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1146\/annurev.bi.61.070192.001515","article-title":"Structure and function of the mannose 6-phosphate\/insulinlike growth factor II receptors","volume":"61","author":"Kornfeld","year":"1992","journal-title":"Annu. Rev. Biochem."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"43160","DOI":"10.1074\/jbc.M107778200","article-title":"Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII","volume":"276","author":"Sands","year":"2001","journal-title":"J. Biol. Chem."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"15172","DOI":"10.1073\/pnas.0607053103","article-title":"Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors","volume":"103","author":"Sly","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1080\/07388551.2020.1869684","article-title":"Characterization of glycosylation in monoclonal antibodies and its importance in therapeutic antibody development","volume":"41","author":"Kaur","year":"2021","journal-title":"Crit. Rev. Biotechnol."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/j.plipres.2014.01.004","article-title":"Ceramide: A simple sphingolipid with unique biophysical properties","volume":"54","author":"Castro","year":"2014","journal-title":"Prog. Lipid Res."},{"key":"ref_40","doi-asserted-by":"crossref","unstructured":"Simonetta, I., Tuttolomondo, A., Daidone, M., and Pinto, A. (2020). Biomarkers in Anderson-Fabry Disease. Int. J. Mol. Sci., 21.","DOI":"10.3390\/ijms21218080"},{"key":"ref_41","first-page":"3733","article-title":"Clinical significance of plasma globotriaosylsphingosine levels in Chinese patients with Fabry disease","volume":"15","author":"Ouyang","year":"2018","journal-title":"Exp. Ther. Med."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.cca.2019.10.031","article-title":"\u03b1-Galactosidase A\/lysoGb3 ratio as a potential marker for Fabry disease in females","volume":"501","author":"Baydakova","year":"2020","journal-title":"Clin. Chim. Acta"},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1016\/j.ymgme.2019.10.010","article-title":"Pathogenesis of Fabry nephropathy: The pathways leading to fibrosis","volume":"129","author":"Rozenfeld","year":"2020","journal-title":"Mol. Genet. Metab."},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"12658","DOI":"10.1073\/pnas.0405042101","article-title":"Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier","volume":"101","author":"Urayama","year":"2004","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/j.jceh.2014.02.005","article-title":"Gaucher disease","volume":"4","author":"Nagral","year":"2014","journal-title":"J. Clin. Exp. Hepatol."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1016\/j.addr.2017.05.004","article-title":"Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives","volume":"118","author":"Solomon","year":"2017","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"76","DOI":"10.2119\/molmed.2011.00248","article-title":"Efficient uptake of recombinant \u03b1-galactosidase A produced with a gene-manipulated yeast by Fabry mice kidneys","volume":"18","author":"Tsukimura","year":"2012","journal-title":"Mol. 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