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Article

Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia.

Human gene therapy - 1 Apr 2011

Chandler Randy J, Chandrasekaran Suma, Carrillo-Carrasco Nuria, Senac Julien S, Hofherr Sean E, Barry Michael A, Venditti Charles P

Abstract excerpt

Propionic acidemia (PA) is an autosomal recessive disorder of metabolism caused by a deficiency of propionyl-coenzyme A carboxylase (PCC). Despite optimal dietary and cofactor therapy, PA patients still suffer from lethal metabolic instability and experience multisystemic complications. A murine model of PA (Pcca(-/-)) of animals that uniformly die within the first 48 hr of life was used to determine the efficacy...

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Source

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Identifiers

PMID: 20950151
DOI: 10.1089/hum.2010.164
PMCID: PMC3073074

Genes

PCCA

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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