| Project/Area Number |
21390270
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Neurology
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| Research Institution | Kumamoto University |
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Principal Investigator |
ANDO Yukio 熊本大学, 大学院・生命科学研究部, 教授 (20253742)
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| Co-Investigator(Kenkyū-buntansha) |
KUME Shoen 熊本大学, 発生医学研究所, 教授 (70347011)
JONO Hirofumi 熊本大学, 大学院・生命科学研究部, 講師 (40515483)
UEDA Mitsuharu 熊本大学, 大学院・生命科学研究部, 助教 (60452885)
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| Co-Investigator(Renkei-kenkyūsha) |
SHIRAKI Nobuaki 熊本大学, 発生医学研究所, 助教 (70448520)
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| Project Period (FY) |
2009 – 2011
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| Project Status |
Completed (Fiscal Year 2011)
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| Budget Amount *help |
¥18,330,000 (Direct Cost: ¥14,100,000、Indirect Cost: ¥4,230,000)
Fiscal Year 2011: ¥2,600,000 (Direct Cost: ¥2,000,000、Indirect Cost: ¥600,000)
Fiscal Year 2010: ¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2009: ¥11,310,000 (Direct Cost: ¥8,700,000、Indirect Cost: ¥2,610,000)
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| Keywords | iPS細胞 / 家族性アミロイドポリニューロパチー / トランスサイレチン / 点変異修復 / 肝細胞置換療法 / アミロイドーシス / FAP / 遺伝子治療 / 肝細胞 / 線維芽細胞 / 移植 |
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| Research Abstract |
From April, 2009 to March, 2012, to develop a curative treatment for familial amyloidotic polyneuropathy(FAP), we obtained somatic cells from FAP patients or transgenic rats possessing a human ATTR V30M gene(ATTR V30M Tg rats) and reprogrammed to pluripotency to generate FAP-specific induced pluripotent stem(iPS) cells, and evaluated the possibility of hepatocyte replacement therapy combining FAP-specific iPS cells and gene-repair therapy using single stranded oligonucleotides(SSOs). In the next project, we will focus on using these FAP-specific iPS cells to elucidate the precise pathogenesis of FAP, improve the efficiency of gene conversion rate, and determine the availability of hepatocyte replacement therapy in the in vivo experiments using ATTR V30M Tg rats, an existing useful FAP animal model.
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