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

Guangyan Wei, Jingsong Cao, Pinzhu Huang, Ping An, Disha Badlani, Kahini A. Vaid, Shuangshuang Zhao, David Q.‐H. Wang, Jenny Zhuo, Ling Yin, Andrea Frassetto, Arianna Markel, Vladimir Presnyak, Srujan Gandham, Serenus Hua, Christine Lukacs, Patrick F. Finn, Paloma H. Giangrande, Paolo G.V. Martini, Yury Popov