Life-Long Correction of Hyperbilirubinemia with a Neonatal Liver-Specific AAV-Mediated Gene Transfer in a Lethal Mouse Model of Crigler–Najjar Syndrome

Null mutations in the UGT1A1 gene result in Crigler–Najjar syndrome type I (CNSI), characterized by severe hyperbilirubinemia and constant risk of developing neurological damage. Phototherapy treatment lowers plasma bilirubin levels, but its efficacy is limited and liver transplantation is required....

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Bibliografiske detaljer
Main Authors: Bortolussi, Giulia, Zentillin, Lorena, Vaníkova, Jana, Bockor, Luka, Bellarosa, Cristina, Mancarella, Antonio, Vianello, Eleonora, Tiribelli, Claudio, Giacca, Mauro, Vitek, Libor, Muro, Andrés F.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2014
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4175423/
https://ncbi.nlm.nih.gov/pubmed/25072305
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.233
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