A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

Crigler-Najjar syndrome is a severe metabolic disease of the liver due to a reduced activity of the UDP Glucuronosyltransferase 1A1 (UGT1A1) enzyme. In an effort to translate to the clinic an adeno-associated virus vector mediated liver gene transfer approach to treat Crigler-Najjar syndrome, we dev...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, Laetitia, Vignaud, Alban, Veron, Philippe, Bosma, Piter J, Muro, Andres F, Mingozzi, Federico
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5052023/
https://ncbi.nlm.nih.gov/pubmed/27722180
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.49
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