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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....

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς: Bortolussi, Giulia, Zentillin, Lorena, Vaníkova, Jana, Bockor, Luka, Bellarosa, Cristina, Mancarella, Antonio, Vianello, Eleonora, Tiribelli, Claudio, Giacca, Mauro, Vitek, Libor, Muro, Andrés F.
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: Mary Ann Liebert, Inc. 2014
Θέματα:
Διαθέσιμο Online: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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