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Article

Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer.

FASEB journal : official publication of the Federation of American Societies for Experimental Biology - 1 Mar 2012

Bortolussi Giulia, Zentilin Lorena, Baj Gabriele, Giraudi Pablo, Bellarosa Cristina, Giacca Mauro, Tiribelli Claudio, Muro Andrés F

Abstract excerpt

Crigler-Najjar type I (CNI) syndrome is a recessively inherited disorder characterized by severe unconjugated hyperbilirubinemia caused by uridine diphosphoglucuronosyltransferase 1A1 (UGT1A1) deficiency. The disease is lethal due to bilirubin-induced neurological damage unless phototherapy is applied from birth. However, treatment becomes less effective during growth, and liver transplantation is required. To...

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Identifiers

PMID: 22094718
DOI: 10.1096/fj.11-195461
PMCID: PMC3370676

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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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