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

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作者： Sem J,Aronson ^[1] ; Robert S,Bakker ^[1] ; Xiaoxia,Shi ^[1] ; Suzanne,Duijst ^[1] ; Lysbeth,Ten Bloemendaal ^[1] ; Dirk R,de Waart ^[1] ; Joanne,Verheij ^[2] ; Giuseppe,Ronzitti ^[3] ; Ronald P,Oude Elferink ^[1] ; Ulrich,Beuers ^[1] ; Coen C,Paulusma ^[1] ; Piter J,Bosma ^[4]

作者单位： Amsterdam University Medical Centers, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, Amsterdam Gastroenterology and Metabolism, Meibergdreef 9, Amsterdam, The Netherlands. ^[1] Amsterdam University Medical Centers, University of Amsterdam, Department of Pathology, Meibergdreef 9, Amsterdam, The Netherlands. ^[2] INTEGRARE, Genethon, INSERM, University of Evry, University Paris-Saclay, 91002 Evry, France. ^[3] Amsterdam University Medical Centers, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, Amsterdam Gastroenterology and Metabolism, Meibergdreef 9, Amsterdam, The Netherlands. Electronic address: p.j.bosma@amc.uva.nl. ^[4]

关键词 AAV AAV8 ABCB4 Adeno-associated virus Correction cholestatic phenotype Gene therapy MDR2 MDR3 PFIC type 3 PFIC3 Progressive familial intrahepatic cholestasis