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Correction of Hyperbilirubinemia in Gunn Rats Using Clinically Relevant Low Doses of Helper-Dependent Adenoviral Vectors

Crigler–Najjar syndrome type I is a severe inborn error of bilirubin metabolism caused by a complete deficiency of uridine diphospho-glucuronosyl transferase 1A1 (UGT1A1) and results in life-threatening unconjugated hyperbilirubinemia. Lifelong correction of hyperbilirubinemia by liver-directed gene...

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Autores principales: Dimmock, David, Brunetti-Pierri, Nicola, Palmer, Donna J., Beaudet, Arthur L., Ng, Philip
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2011
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3073075/
https://ncbi.nlm.nih.gov/pubmed/20973621
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.167
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