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Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4(−/−) mouse model of PFIC3

BACKGROUND & AIMS: Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare lethal autosomal recessive liver disorder caused by loss-of-function variations of the ABCB4 gene, encoding a phosphatidylcholine transporter (ABCB4/MDR3). Currently, no effective treatment exists for PFIC3...

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Detalles Bibliográficos
Autores principales:	Wei, Guangyan, Cao, Jingsong, Huang, Pinzhu, An, Ping, Badlani, Disha, Vaid, Kahini A., Zhao, Shuangshuang, Wang, David Q-H., Zhuo, Jenny, Yin, Ling, Frassetto, Andrea, Markel, Arianna, Presnyak, Vladimir, Gandham, Srujan, Hua, Serenus, Lukacs, Christine, Finn, Patrick F., Giangrande, Paloma H., Martini, Paolo G.V., Popov, Yury V.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8188846/ https://www.ncbi.nlm.nih.gov/pubmed/33340584 http://dx.doi.org/10.1016/j.jhep.2020.12.010

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