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This disease is currently treated primarily by dietary restriction of branched\u2010chain amino acids (BCAAs). However, dietary compliance is often challenging. Conversely, liver transplantation significantly improves outcomes, but donor organs are scarce and there are high costs and potential risks associated with this invasive procedure. Therefore, improved treatment options for MSUD are needed. Development of novel treatments could be facilitated by animal models that accurately mimic the human disease. Animal models provide a useful system in which to explore disease mechanisms and new preclinical therapies. Here we review MSUD and currently available animal models and their corresponding relevance to the human disorder. Using animal models to gain a more complete understanding of the pathophysiology behind the human disease may lead to new or improved therapies to treat or potentially cure the disorder.<\/jats:p>","DOI":"10.1007\/s10545-009-1086-z","type":"journal-article","created":{"date-parts":[[2009,3,5]],"date-time":"2009-03-05T00:23:40Z","timestamp":1236212620000},"page":"229-246","source":"Crossref","is-referenced-by-count":28,"title":["Animal models of maple syrup urine disease"],"prefix":"10.1002","volume":"32","author":[{"given":"K. J.","family":"Skvorak","sequence":"first","affiliation":[{"name":"Graduate Program in the Department of Molecular Genetics and Biochemistry University of Pittsburgh Pittsburgh PA USA"},{"name":"John G. Rangos, Sr. Research Center 530 45th Street, 5th Floor Pittsburgh PA 15201 USA"},{"name":"Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center, Department of Medical Genetics John G. 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