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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 V. Popov

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