Elucidating the RNA pathomechanism in FUS-FALS iPS induced motor neurons

2015 Fiscal Year Final Research Report

• Project/Area Number: 25293199
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Neurology
• Research Institution: Tohoku University
• Principal Investigator: Aoki Masashi 東北大学, 医学(系)研究科(研究院), 教授 (70302148)
• Co-Investigator(Kenkyū-buntansha): Kato MASAAKI 東北大学, 病院, 助教 (50622479) ; Naoki SUZUKI 東北大学, 病院, 助教 (70451599) ; Hitoshi WARITA 東北大学, 大学院医学系研究科, 助教 (30400245)
• Co-Investigator(Renkei-kenkyūsha): OKANO Hideyuki 慶應義塾大学, 医学部, 教授 (60160694)
• Project Period (FY): 2013-04-01 – 2016-03-31
• Keywords: 筋萎縮性側索硬化症 / iPS細胞

Outline of Final Research Achievements

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease resulting in the selective death of motor neurons. ALS symptoms are associated with muscle weakness and paralysis and approximately 80% of ALS patients die within 5 years after the onset of these symptoms. We generated induced pluripotent stem cells (iPSC) from familial ALS (FALS) patients with a missense mutation in the fused-in sarcoma (FUS) gene carrying the heterozygous FUS H517D mutation These cell-derived motor neurons mimicked several neurodegenerative phenotypes including mis-localization of FUS into cytosolic and stress granules under stress conditions, and cellular vulnerability (Ichiyanagi et al. Stem Cell Reports 2016).

Free Research Field

神経内科学