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Article

Long-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.

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

Lee Eun K, Hu Chuhong, Bhargava Ragini, Rozengurt Nora, Stout David, Grody Wayne W, Cederbaum Stephen D, Lipshutz Gerald S

Abstract excerpt

Arginase deficiency is characterized by hyperargininemia and infrequent episodes of hyperammonemia. Human patients suffer from neurological impairment with spasticity, loss of ambulation, seizures, and severe mental and growth retardation. In a murine model, onset of the phenotypic abnormality is heralded by weight loss beginning around day 15 with death occurring typically by postnatal day 17 with...

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Identifiers

PMID: 22760543
DOI: 10.1038/mt.2012.129
PMCID: PMC3464644

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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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