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Article

Liver-directed adenoviral gene transfer in murine succinate semialdehyde dehydrogenase deficiency.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2004

Gupta Maneesh, Jansen Erwin E W, Senephansiri Henry, Jakobs Cornelis, Snead O Carter, Grompe Markus, Gibson K Michael

Abstract excerpt

Murine succinate semialdehyde dehydrogenase (SSADH) deficiency (OMIM 271980; EC 1.2.1.24), a model of the corresponding human disorder, displays 100% mortality at weeks 3-4 of life, associated with lethal tonic-clonic seizures. The biochemical hallmark, gamma-hydroxybutyrate (GHB), accumulates in...

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Identifiers

PMID: 15093183
DOI: 10.1016/j.ymthe.2004.01.013

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026. Prepared record publication-15093183-2026-09-01.1. Manifest publication-graph-cohort-2026-09-01.1.

Source receipt: PubMed via Helena Literature Corpus, retrieved 1 Sept 2026.

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