Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare monogenic disease caused by mutations in the ABCB4 gene, resulting in a reduction in biliary phosphatidylcholine. Reduced biliary phosphatidylcholine cannot counteract the detergent effects of bile salts, leading to cholestasis,...

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Detalles Bibliográficos
Autores principales: Weber, Nicholas D., Odriozola, Leticia, Martínez-García, Javier, Ferrer, Veronica, Douar, Anne, Bénichou, Bernard, González-Aseguinolaza, Gloria, Smerdou, Cristian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6910969/
https://www.ncbi.nlm.nih.gov/pubmed/31836711
http://dx.doi.org/10.1038/s41467-019-13614-3

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