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In mice, the complete lack of CBS is neonatally lethal. In this study, newborn CBS\u2010knockout (KO) mice were treated with recombinant polyethyleneglycolylated human truncated CBS (PEG\u2010CBS). Full survival of the treated KO mice, along with a positive impact on metabolite levels in plasma, liver, brain, and kidneys, was observed. The PEG\u2010CBS treatment prevented an otherwise fatal liver disease characterized by steatosis, death of hepatocytes, and ultrastructural abnormalities of endoplasmic reticulum and mitochondria. Furthermore, treatment of the KO mice for 5 mo maintained the plasma metabolite balance and completely prevented osteoporosis and changes in body composition that characterize both the KO model and human patients. These findings argue that early treatment of patients with HCU with PEG\u2010CBS may prevent clinical symptoms of the disease possibly without the need of dietary protein restriction.\u2014Majtan, T., H\u016flkov\u00e1, H., Park, I., Krijt, J., Ko\u017eich, V., Bublil, E. M., Kraus, J. P. Enzyme replacement prevents neonatal death, liver damage, and osteoporosis in murine homocystinuria. FASEB J. 31, 5495\u20135506 (2017).\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.fasebj.org\">www.fasebj.org<\/jats:ext-link>\n                  <\/jats:p>","DOI":"10.1096\/fj.201700565r","type":"journal-article","created":{"date-parts":[[2017,8,16]],"date-time":"2017-08-16T21:10:34Z","timestamp":1502917834000},"page":"5495-5506","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":29,"title":["Enzyme replacement prevents neonatal death, liver damage, and osteoporosis in murine homocystinuria"],"prefix":"10.1096","volume":"31","author":[{"given":"Tomas","family":"Majtan","sequence":"first","affiliation":[{"name":"Department of Pediatrics University of Colorado School of Medicine Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helena","family":"H\u016flkov\u00e1","sequence":"additional","affiliation":[{"name":"Institute of Inherited Metabolic Disorders and Prague Czech Republic"},{"name":"Institute of Pathology Charles University\u2013First Faculty of Medicine and General University Hospital Prague Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Insun","family":"Park","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Colorado School of Medicine Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jakub","family":"Krijt","sequence":"additional","affiliation":[{"name":"Institute of Inherited Metabolic Disorders and Prague Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Viktor","family":"Ko\u017eich","sequence":"additional","affiliation":[{"name":"Institute of Inherited Metabolic Disorders and Prague Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erez M.","family":"Bublil","sequence":"additional","affiliation":[{"name":"Orphan Technologies Limited Rapperswil Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan P.","family":"Kraus","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Colorado School of Medicine Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2017,8,16]]},"reference":[{"key":"e_1_2_9_2_1","first-page":"2007","volume-title":"In The Metabolic and Molecular Bases of Inherited Disease","author":"Mudd S. 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