Development of novel therapy for progressive familial intrahepatic cholestasis using patient-specific iPS cells

2016 Fiscal Year Final Research Report

• Project/Area Number: 15K19600
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: University of Tsukuba
• Principal Investigator: IMAGAWA Kazuo 筑波大学, 医学医療系, 助教 (40708509)
• Project Period (FY): 2015-04-01 – 2017-03-31
• Keywords: 黄疸 / 遺伝性肝疾患 / iPS細胞

Outline of Final Research Achievements

To develop novel therapy for progressive familial intrahepatic cholestasis type 2, we attempted to establish a PFIC2 model by using iPSC technology. We generated the induced pluripotent stem cells from the patients with progressive familial intrahepatic cholestasis type 2. Then, the induced pluripotent stem cells were differentiated into hepatocyte-like cells. The hepatocyte-like cells expressed albumin, and formed bile canaliculi. The biliary excretion capacity of hepatocyte-like cells was impaired. The biliary excretion capacity of the BD-HLCs could be increased by 4PBA treatment. These results suggested that the drug efficacy could be evaluated by using BD-HLCs.

Free Research Field

小児科学