セッション詳細
[NFS-02]Neuroscience Frontier Symposium 02 Nucleic acid medicine and cellular medicine in the treatment of neurological diseases and the leading edge of research
2025年5月23日(金) 8:00 〜 10:00
第01会場(大阪国際会議場5F 大ホール)
座長：村松 慎一（自治医科大学 神経遺伝子治療部門）, 井上 治久（京都大学iPS細胞研究所）
In 2017, the antisense nucleic acid nusinersen was approved as the first spinal muscular atrophy (SMA) treatment, targeting disease genes for intractable neurological diseases and changing the paradigm of conventional therapeutic thinking. Meanwhile, the era of cellular medicine, in which cells themselves are used as medicines, is currently in progress, driven by the birth of iPS cells. Transplantation therapy of cells for Parkinson's disease and other neurological disorders is about to begin as a real regenerative medicine and cell therapy.
In this symposium, world-class speakers who are trying to conquer intractable neurological diseases for which no curative therapy has been available, with the modality of nucleic acid medicine or cellular medicine, will discuss their research and development.
In this symposium, world-class speakers who are trying to conquer intractable neurological diseases for which no curative therapy has been available, with the modality of nucleic acid medicine or cellular medicine, will discuss their research and development.
[NFS-02-1]Progress of Heteroduplex oligonucleotide (HDO) to muscle and CNS
横田 隆徳 (東京科学大学大学院医歯学総合研究科 脳神経病態学分野（脳神経内科）)
Award:
2001 Award of 42nd Japanese Neurological Association
2003 Award of 56th Japanese Vitamin Association
2006 Award of 18th Meeting of Japanese Society of Neuroimmunology
2021 Award of 33th Meeting of Japanese Society of Neuroimmunology
2022 Award of 18th Meeting of Oligonucleotide Therapeutics Society
2023 The Commendation for Science and Technology by the Minister of Education, Culture, Sports, Science and Technology, Awards for Science and Technology
Extracted Papers:
Mitsugu Yanagidaira, et al. Nat Commun. 2025(in press).
Hasegawa J, et al. Nat Commun. 2024;15:7530.
Ohyagi M, et al. Nat Commun. 2021;12:7344.
Nishina K, et al. Nat Commun. 2015;6:7969.
Sasaguri H, et al. Nat Commun. 2018;9:2892.
2001 Award of 42nd Japanese Neurological Association
2003 Award of 56th Japanese Vitamin Association
2006 Award of 18th Meeting of Japanese Society of Neuroimmunology
2021 Award of 33th Meeting of Japanese Society of Neuroimmunology
2022 Award of 18th Meeting of Oligonucleotide Therapeutics Society
2023 The Commendation for Science and Technology by the Minister of Education, Culture, Sports, Science and Technology, Awards for Science and Technology
Extracted Papers:
Mitsugu Yanagidaira, et al. Nat Commun. 2025(in press).
Hasegawa J, et al. Nat Commun. 2024;15:7530.
Ohyagi M, et al. Nat Commun. 2021;12:7344.
Nishina K, et al. Nat Commun. 2015;6:7969.
Sasaguri H, et al. Nat Commun. 2018;9:2892.
[NFS-02-2]Silencing FUS and Other Rare Genetic Forms of ALS
Neil A. Shneider (Columbia University)
Dr. Shneider is the Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University. He is an investigator in the Center for Motor Neuron Biology and Disease where his lab focuses on the study of models and mechanisms of ALS and the development of novel therapeutics for ALS and related disorders. Dr. Shneider worked with Ionis Pharmaceuticals to develop ION363 (jacifusen), an antisense oligonucleotide (ASO) for ALS patients with rare mutations in the FUsed in Sarcoma (FUS) gene. Dr. Shneider is a graduate of Harvard College and earned his M.D. and Ph.D. degrees at the Columbia University College of Physicians and Surgeons. In partnership with n-Lorem and Columbia University, Dr. Shneider founded Silence ALS, an initiative to develop ASOs for ALS patients with ultra-rare, pathogenic mutations in ALS genes. Dr. Shneider was co-chair of the Translating Fundamental Research into Potential ALS Therapies Working Group for the NIH ALS Strategic Planning Workshop, and is a multi-PI for the Access for ALL in ALS Consortium (ALL ALS), a community of 35 research sites across the United States conducting a combined longitudinal natural history study and biomarker collection study for ALS.
[NFS-02-3]ミクログリア置換による神経変性疾患の治療
小泉 修一1,2 (1.山梨大学 医学部薬理学講座, 2.山梨GLIAセンター)
1992: Ph.D., Kyushu Univ, Fukuoka, Japan
1992-1995: Posdoc, National Institute of Health Sciences (NIHS), Tokyo, Japan
1996-1999: Posdoc, Univ Cambridge, Cambridge, UK. (Prof. Mike Berridge)
1999-2003: Researcher, NIHS, Tokyo
2003-2006: Senior Researcher, NIHS, Tokyo
2007-present: Professor, Dept Neuropharmacol, Grad Sch Med, Univ Yamanashi, Japan
2017-2021: Vice Dean, Facult Med, Univ Yamanashi
2021-present: Director, Yamanashi GLIA Center
2023-present: Dean, Facult Med, Univ Yamanashi
1992-1995: Posdoc, National Institute of Health Sciences (NIHS), Tokyo, Japan
1996-1999: Posdoc, Univ Cambridge, Cambridge, UK. (Prof. Mike Berridge)
1999-2003: Researcher, NIHS, Tokyo
2003-2006: Senior Researcher, NIHS, Tokyo
2007-present: Professor, Dept Neuropharmacol, Grad Sch Med, Univ Yamanashi, Japan
2017-2021: Vice Dean, Facult Med, Univ Yamanashi
2021-present: Director, Yamanashi GLIA Center
2023-present: Dean, Facult Med, Univ Yamanashi
[NFS-02-4]脳腫瘍に対する遺伝子幹細胞療法
戸田 正博 (慶應義塾大学医学部 脳神経外科)
Masahiro Toda, MD, PhD
Professor and Chairman, Department of Neurosurgery, Keio University School of Medicine
1987 M.D. Keio University School of Medicine
1995 Research Fellow, Department of Neurosurgery, Georgetown University Medical Center
1997 Instructor, Department of Physiology, Keio University School of Medicine
1999 Instructor, Institute for Advanced Medical Research, Keio University School of Medicine
2012 Associate Professor, Department of Neurosurgery, Keio University School of Medicine
2020 Visiting professor, Department of Neurosurgery, Faculty of Medicine University of Padjadjaran
2020 Professor, Department of Neurosurgery, Keio University School of Medicine
Societies:
Director, The Japan Neurosurgical Society
President, Japanese Society for Neuroendoscopy
Director, Japan Society for Pituitary and Hypothalamic Tumor
Director, The Japanese Congress for Brain Tumor Surgery
Director, Japanese Society for Skull Base Surgery
Director, Japan Society of Gene and Cell Therapy
Professor and Chairman, Department of Neurosurgery, Keio University School of Medicine
1987 M.D. Keio University School of Medicine
1995 Research Fellow, Department of Neurosurgery, Georgetown University Medical Center
1997 Instructor, Department of Physiology, Keio University School of Medicine
1999 Instructor, Institute for Advanced Medical Research, Keio University School of Medicine
2012 Associate Professor, Department of Neurosurgery, Keio University School of Medicine
2020 Visiting professor, Department of Neurosurgery, Faculty of Medicine University of Padjadjaran
2020 Professor, Department of Neurosurgery, Keio University School of Medicine
Societies:
Director, The Japan Neurosurgical Society
President, Japanese Society for Neuroendoscopy
Director, Japan Society for Pituitary and Hypothalamic Tumor
Director, The Japanese Congress for Brain Tumor Surgery
Director, Japanese Society for Skull Base Surgery
Director, Japan Society of Gene and Cell Therapy
[NFS-02-5]iPS細胞を用いたパーキンソン病治療
髙橋 淳 (京都大学ｉＰＳ細胞研究所 臨床応用研究部門)
Jun Takahashi is a professor and director of the Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan. He graduated from the Kyoto University Faculty of Medicine in 1986 and thereafter started his career as a neurosurgeon at Kyoto University Hospital. After he earned his Ph.D. from the Kyoto University Graduate School of Medicine, he worked as a postdoctoral research fellow at the Salk Institute (Dr. Fred Gage), CA, U.S.A., where he started research work on neural stem cells. After returning to Kyoto University Hospital, he conducted functional neurosurgery including deep brain stimulation and also research work on stem cell therapy for Parkinson’s disease. In 2012, he became a full professor at CiRA, pursuing stem cell therapies for Parkinson’s disease patients. As a physician-scientist, he has laid the groundwork for the clinical application of iPS cells and started the world’s first clinical trial for Parkinson’s disease using iPS cells in 2018.