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Article

Critical assessment of lifelong phenotype correction in hyperbilirubinemic Gunn rats after retroviral mediated gene transfer.

Gene therapy - 1 Sept 2007

Nguyen T H, Aubert D, Bellodi-Privato M, Flageul M, Pichard V, Jaidane-Abdelghani Z, Myara A, Ferry N

Abstract excerpt

Among inherited diseases of the liver, Crigler-Najjar type 1 disease (CN-1), which results from complete deficiency in bilirubin UDP-glucuronosyltransferase activity (B-UGT1), is an attractive target for gene therapy studies. Hyperbilirubinemic Gunn rats, a model of CN-1, were injected at 2 days of age with lentiviral or oncoretroviral vectors encoding the human B-UGT1. After injection, bilirubinemia was...

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Identifiers

PMID: 17611583
DOI: 10.1038/sj.gt.3302993

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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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