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Article

Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jun 2012

Keeler Allison M, Conlon Thomas, Walter Glenn, Zeng Huadong, Shaffer Scott A, Dungtao Fu, Erger Kirsten, Cossette Travis, Tang Qiushi, Mueller Christian, Flotte Terence R

Abstract excerpt

Very long-chain acyl-coA dehydrogenase (VLCAD) is the rate-limiting step in mitochondrial fatty acid oxidation. VLCAD-deficient mice and patients clinical symptoms stem from not only an energy deficiency but also long-chain metabolite accumulations. VLCAD-deficient mice were treated systemically with 1 × 10(12) vector genomes of recombinant adeno-associated virus 9 (rAAV9)-VLCAD. Biochemical correction was...

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Identifiers

PMID: 22395529
DOI: 10.1038/mt.2012.39
PMCID: PMC3370259

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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