Gloria Gonzalez-Aseguinolaza

Liver-directed gene therapy results in amelioration of progressive familial intrahepatic cholestasis type 2 in mice

Background: Progressive familial intrahepatic cholestasis type 2 (PFIC2) is a rare disease affecting the ABCB11 gene, encoding the bile salt export pump (BSEP). BSEP dysfunction impairs bile acid (BA) secretion, causing hepatic damage and leading to pruritus, cholestasis, hepatom...