Hepatocyte Transplantation Improves Phenotype and Extends Survival in a Murine Model of Intermediate Maple Syrup Urine Disease

Maple syrup urine disease (MSUD; OMIM 248600) is an inborn error of metabolism of the branched chain α-ketoacid dehydrogenase (BCKDH) complex that is treated primarily by dietary manipulation of branched-chain amino acids (BCAA). Dietary restriction is lifelong and compliance is difficult. Liver tra...

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Main Authors: Skvorak, Kristen J, Paul, Harbhajan S, Dorko, Kenneth, Marongiu, Fabio, Ellis, Ewa, Chace, Donald, Ferguson, Carolyn, Gibson, K Michael, Homanics, Gregg E, Strom, Stephen C
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2009
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Original Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835204/
https://ncbi.nlm.nih.gov/pubmed/19436271
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.99
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