Therapeutic strategy of neurodegenerative disease using microRNA

Research Project

Project/Area Number	25670418
Research Category	Grant-in-Aid for Challenging Exploratory Research
Allocation Type	Single-year Grants
Research Field	Neurology
Research Institution	Nagoya University
Principal Investigator	SOBUE Gen 名古屋大学, 医学(系)研究科(研究院), 教授 (20148315)
Co-Investigator(Kenkyū-buntansha)	ADACHI Hiroaki 名古屋大学, 大学院医学系研究科, 寄附講座准教授 (40432257) KATSUNO Masahisa 名古屋大学, 大学院医学系研究科, 准教授 (50402566)
Project Period (FY)	2013-04-01 – 2014-03-31
Project Status	Completed (Fiscal Year 2013)
Budget Amount *help	¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000) Fiscal Year 2013: ¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000)
Keywords	脳神経疾患 / トランスレーショナルリサーチ / 遺伝子 / 核酸 / 発現制御
Research Abstract	We identified RNA-binding proteins that influence most strongly on the stability of the mRNA of Neuropathology spinocerebellar degeneration and (HD) Huntington's disease (SCD) in cultured cell experiments. In cell culture experiments, I found that CUGBP is an RNA-binding protein, Elav-like family (CELF) suppresses the mRNA expression of pathogenesis of SCD gene and HD. In addition, this expression suppression effect was not independent of the length of the array length of the gene-specific pathogenesis of SCD and HD in (CAG repeat number). Currently, it is administered to a mouse model of SCD and HD prepared and elucidation of the mechanism by which CELF proteins affect the expression of mRNA of pathogenesis gene of SCD and HD, the AAV expressing the CELF proteins, we examined the therapeutic effect.