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

doi:10.1016/j.jhep.2020.12.010

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Article

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

Journal of hepatology - 1 Jun 2021

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

Abstract excerpt

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 outside of liver transplantation. METHODS: We have produced and screened chemically and genetically modified mRNA...

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Identifiers

PMID: 33340584
DOI: 10.1016/j.jhep.2020.12.010
PMCID: PMC8188846

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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