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Article

Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery.

Gene therapy - 1 Apr 2012

Sénac J S, Chandler R J, Sysol J R, Li L, Venditti C P

Abstract excerpt

Methylmalonic acidemia (MMA), an inherited metabolic disorder caused by deficient activity of methylmalonyl-CoA mutase, carries a poor prognosis for long-term survival. While administration of a recombinant adeno-associated virus serotype 8 vector (rAAV8) can rescue Mut(-/-) mice from neonatal lethality and provide sustained phenotypic correction, translation of gene therapy to human subjects will likely require...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 21776024
DOI: 10.1038/gt.2011.108
PMCID: PMC3382069

Provenance

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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