Development of novel therapeutic method for ABC transporter-related diseases
| Project/Area Number |
26460192
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Medical pharmacy
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| Research Institution | The University of Tokyo |
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Principal Investigator |
HAYASHI Hisamitsu 東京大学, 大学院薬学系研究科(薬学部), 助教 (10451858)
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| Project Period (FY) |
2014-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| 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)
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| Keywords | ABCトランスポーター / 胆汁うっ滞症 / タンパク質分解 / 胆汁うっ滞 |
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| 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.
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Report
(4 results)
Research Products
(25 results)
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[Journal Article] Effects of 4-phenylbutyrate therapy in a preterm infant with cholestasis and liver fibrosis2016
Author(s)
Ito S, Hayashi H, Sugiura T, Ito K, Ueda H, Togawa T, Endo T, Tanikawa K, Kage M, Kusuhara H, Saitoh S.
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Journal Title
Pediatr Int.
Volume: Feb 4
Issue: 6
Pages: 506-509
DOI
Related Report
Peer Reviewed
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[Journal Article] Improved Liver Function and Relieved Pruritus after 4-Phenylbutyrate Therapy in a Patient with Progressive Familial Intrahepatic Cholestasis Type 2.2014
Author(s)
Naoi S, Hayashi H, Inoue T, Tanikawa K, Igarashi K, Nagasaka H, Kage M, Takikawa H, Sugiyama Y, Inui A, Nagai T, Kusuhara H.
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Journal Title
Journal of Pediatrics
Volume: 164
Issue: 5
Pages: 1219-1227
DOI
Related Report
Peer Reviewed
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[Journal Article] Intractable itch relieved by 4-phenylbutyrate therapy in patients with progressive familial intrahepatic cholestasis type 1.2014
Author(s)
Hasegawa Y, Hayashi H, Naoi S, Kondou H, Bessho K, Igarashi K, Hanada K, Nakao K, Kimura T, Konishi A, Nagasaka H, Miyoshi Y, Ozono K, Kusuhara H
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Journal Title
Orphanet J Rare Dis.
Volume: 9
Issue: 1
Pages: 89-89
DOI
Related Report
Peer Reviewed / Open Access
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