Disease mechanism research using cellular amyotrophic lateral sclerosis models derived from hES cells

2013 Fiscal Year Final Research Report

• Project/Area Number: 23500446
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Neurochemistry/Neuropharmacology
• Research Institution: Kyoto University
• Principal Investigator: AIBA Kazuhiro 京都大学, 物質-細胞統合システム拠点, 准教授 (30564752)
• Project Period (FY): 2011 – 2013
• Keywords: 筋萎縮性側索硬化症 / 脳神経疾患 / ヒトES細胞 / ヒト多能性幹細胞 / 疾患モデル細胞

Research Abstract

Overexpression of SOD1, a responsible gene of amyotrophic lateral sclerosis (ALS) did not affect differentiation of motor neuron and astrocyte from human embryonic stem cells. But we found that one mutant type of SOD1 affected the length of neuritis. In future, we will investigate whether this change is involved in the onset or progress of ALS.

Research Products

• [Journal Article] Multidisciplinary research of human pluripotent stem cells for application to cell therapy and drug discovery (2013)
  • Author(s): Nakatsuji N, Kawase E, Miyazaki T, Minami I, Aiba K
  • Volume: 10
  • Pages: 160-163
  • DOI: 10.1007/s13770-013-0019-y
• [Journal Article] Amyotrophic Lateral Sclerosis Model Derived from Human Embryonic Stem Cells Overexpressing Mutant Superoxide Dismutase 1 (2012)
  • Author(s): Wada T, Goparaju SK, Tooi N, Inoue H, Takahashi R, Nakatsuji N, Aiba K
  • Journal Title: Stem Cells Trans Med Volume: 1 Pages: 396-402
  • DOI: 10.5966/sctm.2011-0061
  • Peer Reviewed
• [Journal Article] Chemical library screening identifies a small molecule that downregulates SOD1 transcription for drugs to treat amyotrophic lateral sclerosis (2011)
  • Author(s): Murakami G, Inoue H, Tsukita K, Asai Y, Amagai Y, Aiba K, Shimogawa H, Uesugi M, Nakatsuji N, Takahashi R
  • Volume: 11
  • Pages: 405-414
  • DOI: 10.1177/1087057110397888
  • Peer Reviewed
• [Presentation] 遺伝子改変ヒトES 細胞由来ALS-SOD1 モデル細胞の作製および解析 (2014)
  • Author(s): 磯部武久, 遠井紀江, 中辻憲夫, 饗庭一博
  • Organizer: 日本薬学会第134回年会
  • Place of Presentation: 熊本県熊本市
  • Year and Date: 2014-03-30
• [Presentation] 遺伝子改変ヒトES 細胞由来ALS モデル細胞の作製および病態解析 (2013)
  • Author(s): 磯部武久, 遠井紀江, 饗庭一博, 中辻憲夫
  • Organizer: 第36回日本分子生物学会年会
  • Place of Presentation: 兵庫県神戸市
  • Year and Date: 2013-12-04
• [Presentation] Amyotrophic lateral sclerosis model cells derived from human embryonic stem cells overexpressing mutant SOD1 or TDP-43 (2013)
  • Author(s): Isobe T, Tooi N, Aiba K, Nakatsuji N
  • Organizer: ISSCR 11th Annual meeting
  • Place of Presentation: アメリカ合衆国ボストン
  • Year and Date: 2013-06-13
• [Presentation] Cellular neurodegenerative disease models derived from genetically manipulated human pluripotent stem cells (2012)
  • Author(s): Aiba K, Honda M, Goparaju SK, Tooi N, Minami I, Nakatsuji N
  • Organizer: ISSCR 10th Annual Meeting
  • Place of Presentation: 神奈川県横浜市
  • Year and Date: 20120613-16
• [Presentation] Development of a human embryonic stem cell-based disease model for amyotrophic lateral sclerosis (2012)
  • Author(s): Goparaju SK, Wada T, Tooi N, Aiba K, Nakatsuji N
  • Organizer: ISSCR 10th Annual Meeting
  • Place of Presentation: 神奈川県横浜市
  • Year and Date: 20120613-16
• [Presentation] Chemical library screening identifies a small molecule that downregulates SOD1 transcription for drugs to treat amyotrophic lateral sclerosis (2012)
  • Author(s): Murakami G, Inoue H, Asai Y, Aiba K, Itoh H, Uesugi M, Nakatsuji N, Takahashi R
  • Organizer: 13th Asian Oceanian Congress of Neurology
  • Place of Presentation: オーストラリア メルボルン
  • Year and Date: 20120603-08
• [Presentation] Site-directed integration of transgenes and human pluripotent stem cell-derived neurodegenerative disease models (2012)
  • Author(s): Aiba K
  • Organizer: Using Stem Cells for Biological and Therapeutical Discovery in Mental Illness
  • Place of Presentation: アメリカ合衆国ベセスダ
  • Year and Date: 2012-04-25
• [Presentation] ヒト多能性幹細胞から作製されるモデル細胞-神経変性疾患モデルと心筋モデル- (2012)
  • Author(s): 饗庭一博
  • Organizer: 日本農芸化学会
  • Place of Presentation: 京都府京都市
  • Year and Date: 2012-03-23
• [Book] Cre/loxP, Flp/FRT systems and pluripotent stem cell lines. In Site-directed insertion of transgenes (2012)
  • Author(s): Tahimic CGT, Sakurai K, Aiba K, and Nakatsuji N
  • Total Pages: 189-209
  • Publisher: Springer
• [Remarks] ホームページ
  • URL: http://www.icems.kyoto-u.ac.jp/j/ppl/grp/nakatsuji.html
• [Remarks] ヒトES 細胞からALS 疾患モデルを作製し, 病状再現に成功, NHK(5月9日, 10日), 京都新聞(5月9日 1面), 産経新聞(5月9日 2面), 読売新聞(5月9日 33 面), 朝日新聞(5月9日 夕刊 8面),日本経済新聞(5月9日 34面),日刊工業新聞(5月9日 19 面), Wall Street Journal日本版(2012年5月9日 web), その他 (2012年5月) (報道)
  • URL: http://www.icems.kyoto-u.ac.jp/j/pr/2012/05/09-nr.html