Cyclodextrins as Modifiers of Intracellular Cholesterol Trafficking in Niemann-Pick Disease Type C
| Project/Area Number |
23590642
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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 |
Applied pharmacology
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| Research Institution | Kumamoto University |
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Principal Investigator |
IRIE Tetsumi 熊本大学, 大学院生命科学研究部, 教授 (60150546)
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| Co-Investigator(Kenkyū-buntansha) |
ISHITSUKA Yoichi 熊本大学, 生命科学研究部, 講師 (70423655)
ARIMA Hidetoshi 熊本大学, 生命科学研究部, 教授 (50260964)
MOTOYAMA Keiichi 熊本大学, 生命科学研究部, 准教授 (50515608)
MATSUO Muneaki 佐賀大学, 医学部, 准教授 (20219398)
MOCHINAGA Sakiko 佐賀大学, 医学部, 医療技術職員 (70601045)
FUJITO Hiroshi 佐賀大学, 医学部, 教授 (50325594)
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| Project Period (FY) |
2011 – 2013
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| Project Status |
Completed (Fiscal Year 2013)
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| Budget Amount *help |
¥5,200,000 (Direct Cost: ¥4,000,000、Indirect Cost: ¥1,200,000)
Fiscal Year 2013: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2012: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2011: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
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| Keywords | Niemann-Pick Type C病 / シクロデキストリン / ニーマン・ピック病C型 |
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| Research Abstract |
2-Hydroxypropyl-beta-cyclodextrin (HPBCD) is an attractive drug candidate against Niemann-Pick disease Type C (NPC). However, the efficacy and safety of HPBCD treatment for NPC patients remain to be elucidated. This study was conducted to obtain the essential information on efficacy and safety of HPBCD treatment for NPC patients. 1) Weekly subcutaneous administrations of HPBCD at a dose of 1000-4000 mg/kg to Npc1-deficient mice ameliorated hepatomegaly and survival rates in the mice. 2) Compared with wild type mice, Npc1-deficient mice were substantially resistant to the lethality and the organ injury induced by HPBCD at higher doses. This study provides information on optimization of HPBCD treatment for NPC patients and also suggests Npc1 genotype should be considered in the safety evaluation of HPBCD using animals and cells.
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Report
(4 results)
Research Products
(65 results)
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[Journal Article] Influence of Npc1 genotype on the toxicity of hydroxypropyl -β-cyclodextrin, a potentially therapeutic agent, in Niemann-Pick Type C disease models2014
Author(s)
Tanaka Y, Ishitsuka Y, Yamada Y, Kondo Y, Takeo T, Nakagata N, Higashi T, Motoyama K, Arima H, Matsuo M, Higaki K, Ohno K, Irie T
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Journal Title
Molecular Genetics and Metabolism Reports
Volume: 1
Pages: 31-41
DOI
Related Report
Peer Reviewed
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[Journal Article] Comparative effects of phosphoenolpyruvate (PEP), a glycolytic intermediate, as an organ preservation agent with glucose and N-acetylcysteine against organ damage during cold storage of mouse liver and kidney2013
Author(s)
Ishitsuka Y, Fukumoto Y, Kondo Y, Irikura M, Kadowaki D, Narita Y, Hirata S, Moriuchi H, Maruyama T, Hamasak N, Irie T
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Journal Title
ISRN Pharmacology
Volume: 2013
Pages: 375825-375825
DOI
Related Report
Peer Reviewed
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[Journal Article] Phosphoenolpyruvic acid (PEP), an intermediary metabolite of glycolysis, as a potential cytoprotectant and anti-oxidant in HeLa cells2012
Author(s)
Kondo Y and Ishitsuka Y, Kadowaki D, Kuroda M, Tanaka Y, Nagatome M, Irikura M, Hirata S, Sato K, Maruyama T, Hamasaki N, and Irie T
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Journal Title
Biological and Pharmaceutical Bulletin
Volume: 35
Pages: 606-611
Related Report
Peer Reviewed
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[Presentation] Antiproliferative effect of 2-hydroxypropyl-β-cyclodextrin (HP-β-CyD) against chronic myeloid leukemia in vitro and in vivo2012
Author(s)
Masako Yokoo, Yasushi Kubota, Sakiko Mochinaga, Aya Maeda, Tatsuo Ichinohe, Hiroshi Fujito, Taishi Higashi, Keiichi Motoyama, Hidetoshi Arima, Tetsumi Irie, Shinya Kimura
Organizer
54th ASH Annual Meeting and Exposition
Place of Presentation
USA, Atlanta, Georgia World Congress Center
Year and Date
2012-12-08
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