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

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 appli...

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Detalles Bibliográficos
Autores principales:	Bortolussi, Giulia, Zentilin, Lorena, Baj, Gabriele, Giraudi, Pablo, Bellarosa, Cristina, Giacca, Mauro, Tiribelli, Claudio, Muro, Andrés F.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Federation of American Societies for Experimental Biology 2012
Materias:	Research Communications
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3370676/ https://www.ncbi.nlm.nih.gov/pubmed/22094718 http://dx.doi.org/10.1096/fj.11-195461

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