Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
Abstract
Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare monogenic disease caused by mutations in the ABCB4 gene, resulting in a reduction in biliary phosphatidylcholine. Reduced biliary phosphatidylcholine cannot counteract the detergent effects of bile salts, leading to cholestasis, cholangitis, cirrhosis and ultimately liver failure. Here, we report results from treating two- or five-week-old Abcb4 −/− mice with an AAV vector...
Paper Details
Title
Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
Published Date
Dec 13, 2019
Journal
Volume
10
Issue
1
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