Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models

＜Dissertation information＞
Journal：Molecular Therapy Advances
Title：Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models
DOI:10.1016/j.omta.2026.201835
Author：Keiko Imamura^1,2,3, Shin Yoshioka⁴, Kota Kamizato⁵, Ryosuke Oki⁶, Hiroyuki Hioki⁷, Kayoko Tsukita^1,2, Ikuyo Inoue^1,3, Rina Shimizu^1,3, Mako Takiguchi^1,3, Tomoki Sakasai¹, Aya Okusa¹, Ran Shibukawa², Takeshi Niki^1,2, Satoko Sakurai¹, Taro Okunomiya^1,2, Takayuki Kondo^1,2,3, Tomohisa Kato Jr^8,9, Atsushi Miyanohara¹⁰, Manabu Kakinohana⁵, Takuya Yamamoto^1,3,11, Martin Marsala¹⁰, Keiji Nishida^4,12, Yuishin Izumi^6,*, Haruhisa Inoue^1,2,3,*

1 　 Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan
2 　 iPSC-based Drug Discovery and Development Team, RIKEN BioResource Research Center (BRC), Kyoto, Japan
3 　 Medical-Risk Avoidance based on iPS Cells Team, RIKEN Center for Advanced Intelligence Project (AIP), Kyoto, Japan
4 　 Graduate School of Science, Technology and Innovation, Kobe University, Hyogo, Japan
5 　 Department of Anesthesiology, Graduate School of Medicine, University of the Ryukyus, Okinawa, Japan
6 　 Department of Neurology, Tokushima University Graduate School of Biomedical Sciences, Tokushima, Japan
7 　 Department of Neuroanatomy, Juntendo University Graduate School of Medicine, Tokyo, Japan
8 　 Division of iPS Cell Applied Medicine, Department of Advanced Medicine, Medical Research Institute, Kanazawa Medical University, Ishikawa, Japan
9 　 Center for Regenerative Medicine, Kanazawa Medical University Hospital, Ishikawa, Japan
10　Neuroregeneration Laboratory, Department of Anesthesiology, University of California San Diego, La Jolla, CA, USA
11　Institute for the Advanced Study of Human Biology (WPI-ASHBi), Kyoto University, Kyoto, Japan
12　Engineering Biology Research Center, Kobe University, Hyogo, Japan