However, several preclinical studies have provided the proof of concept of the feasibility of this strategy in animal models of PFIC3.[25], [26], [27], [28] This strategy bears the highest hope in treating patients with PFIC3, especially those with a severe genotype and/or a negative response to UDCA for whom the only currently available treatment is LT. Here, ABCB4 is linked to progressive familial intrahepatic cholestasis type 3.