| Budget Amount *help |
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2016: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2015: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2014: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
|
|---|
| Outline of Final Research Achievements |
Progressive familial intrahepatic cholestasis type 2 (PFIC2), a pediatric liver disease, is caused by mutations in ABCB11 encoding BSEP, an ABC transporter. No effective medical therapy has been established for PFIC2. Based on our previous findings from in vitro and animal studies, in this research project, we carried out clinical study to evaluate efficacy and safety of sodium phanylbutyrate (NaPB) in patients with PFIC2. Therapy with NaPB significantly improved biochemical tests and liver histology in the patients. Because this effect of NaPB involved inhibition of BSEP degradation, molecular mechanism underlying the degradation of BSEP and other ABC transporters was studied. A couple of molecules responsible for this mechanism have been identified.
|
