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Enzyme replacement therapy (ERT<\/jats:styled-content>) is the current treatment for MPS VI<\/jats:styled-content>, although it involves limited compliance to the therapy and high cost. The aim of this study was to develop a new method of treatment by conducting an orthotopic liver transplantation (LT<\/jats:styled-content>x) using an animal model of human MPS VI<\/jats:styled-content>, and to evaluate and examine its effectiveness for treating MPS VI<\/jats:styled-content>.<\/jats:p><\/jats:sec>Methods<\/jats:title>LT<\/jats:styled-content>x was carried out from normal unaffected to affected MPS VI<\/jats:styled-content> rats (MPR<\/jats:styled-content>), which were then killed after LT<\/jats:styled-content>x, and tissues from the heart, spleen, and knee joint, as well as serum, collected for biological and morphologic evaluation.<\/jats:p><\/jats:sec>Results<\/jats:title>Liver\u2010transplanted (LT<\/jats:styled-content>x) MPR<\/jats:styled-content> had the same level of N<\/jats:italic>\u2010acetylgalactosamine 4\u2010sulfatase activity in the liver and lungs as normal unaffected MPR<\/jats:styled-content>, and the urinary secretion of mucopolysaccharides\/glycosaminoglycan (GAG<\/jats:styled-content>) in LT<\/jats:styled-content>x MPR<\/jats:styled-content> was significantly decreased. Furthermore, on histopathology, the spleens of LT<\/jats:styled-content>x MPR<\/jats:styled-content> showed elimination of vacuole cells. In the knee joints, growth plates became thinner, and on radiography the facial and cranial bones of LT<\/jats:styled-content>x MPR<\/jats:styled-content> were morphologically normal.<\/jats:p><\/jats:sec>Conclusions<\/jats:title>LT<\/jats:styled-content>x from normal to affected MPR<\/jats:styled-content> was effective for symptoms of MPS<\/jats:styled-content> and accumulation of GAG<\/jats:styled-content>, suggesting that LT<\/jats:styled-content>x could be a promising alternative approach for MPS VI<\/jats:styled-content>.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ped.13751","type":"journal-article","created":{"date-parts":[[2018,12,13]],"date-time":"2018-12-13T22:00:43Z","timestamp":1544738443000},"page":"180-189","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Liver transplantation: New treatment for mucopolysaccharidosis type VI<\/scp> in 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