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Article

Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1.

Gene therapy - 1 Dec 2013

Chandler R J, Tarasenko T N, Cusmano-Ozog K, Sun Q, Sutton V R, Venditti C P, McGuire P J

Abstract excerpt

Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life-threatening episodes of acute hyperammonemia. A murine model of CTLN1 (fold/fold) that displays lethality within the first 21 days of life was used to determine the...

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Source

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Identifiers

PMID: 24131980
DOI: 10.1038/gt.2013.53
PMCID: PMC3855546

Genes

ASS1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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