Liver-directed gene therapy results in long term correction of progressive familial intrahepatic cholestasis type 3 in mice.

•Adeno-associated virus (AAV)-mediated gene therapy can correct Abcb4 deficiency (PFIC3) in mice.•By restoring phospholipid transport to bile, cholestasis and liver damage were strongly reduced.•Stable transgene expression resulted in long-term correction of the phenotype (26 weeks).•Hepatic transgene persistence was achieved by sufficiently reducing hepatocyte proliferation.