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Article

Hepatocyte transplantation improves phenotype and extends survival in a murine model of intermediate maple syrup urine disease.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jul 2009

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

Abstract excerpt

Maple syrup urine disease (MSUD; OMIM 248600) is an inborn error of metabolism of the branched chain alpha-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. Li...

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Source

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Identifiers

PMID: 19436271
DOI: 10.1038/mt.2009.99
PMCID: PMC2835204

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-19436271-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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