Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV‐piggyBac Gene Therapy
Susan M. Siew, Sharon C. Cunningham, Erhua Zhu, Szun S. Tay, Elena Venuti, Christine Bolitho, Ian E. Alexander – 17 May 2019 – Recombinant adeno‐associated viral (rAAV) vectors are highly promising vehicles for liver‐targeted gene transfer, with therapeutic efficacy demonstrated in preclinical models and clinical trials. Progressive familial intrahepatic cholestasis type 3 (PFIC3), an inherited juvenile‐onset, cholestatic liver disease caused by homozygous mutation of the ABCB4 gene, may be a promising candidate for rAAV‐mediated liver‐targeted gene therapy.