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

Overview

Authors: Keiko Imamura1,2,3, Shin Yoshioka4, Kota Kamizato5, Ryosuke Oki6, Hiroyuki Hioki7, Kayoko Tsukita1,2, Ikuyo Inoue1,3, Rina Shimizu1,3, Mako Takiguchi1,3, Tomoki Sakasai1, Aya Okusa1, Ran Shibukawa2, Takeshi Niki1,2, Satoko Sakurai1, Taro Okunomiya1,2, Takayuki Kondo1,2,3, Tomohisa Kato Jr8,9, Atsushi Miyanohara10, Manabu Kakinohana5, Takuya Yamamoto1,3,11, Martin Marsala10, Keiji Nishida4,12, Yuishin Izumi6, Haruhisa Inoue1,2,3
ORCID iDs: Yuishin Izumi, Haruhisa Inoue
  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
Institutions: Kyoto University (Japan); RIKEN BioResource Research Center (Japan); RIKEN Center for Advanced Intelligence Project (Japan); Center for iPS Cell Research and Application, Kyoto University (Japan); Kobe University (Japan); University of the Ryukyus (Japan); Tokushima University (Japan); Juntendo University (Japan); Kanazawa Medical University (Japan); Kanazawa Medical University Hospital (Japan); University of California San Diego (United States)
Journal: Molecular therapy. Advances, volume 34, issue 4, article 201835
Dates: received 9 March 2026; accepted 19 August 2026; published online 21 August 2026
Type: Research article · Language: English
License: CC BY-NC-ND
Identifiers: DOI 10.1016/j.omta.2026.201835 · PMID 42740795 · PMCID PMC13571996 · OpenAlex W7203861736
Open access: hybrid, a free copy (OpenAlex)
Status: code on request
Categories: human (organism), mouse (organism), other condition (population)
Methods: Statistics, fMRI & imaging
Keywords: gene therapy, base editing therapy, ABE, iPSCs, organoids, subpial injection, AAV, ALS, MND, HMSN-P
Topic: Amyotrophic Lateral Sclerosis Research (Neurology, Medicine), according to OpenAlex
Funding: Japan Agency for Medical Research and Development
Citations: not cited yet (Europe PMC); 43 references in the paper
Research resources: RRID:Addgene_138495

Abstract

The abstract is not reproduced here: the paper's license (CC BY-NC-ND) does not allow it. Read it in the paper, at the publisher or on Europe PMC.

Code

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Data

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Code and data availability statement

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Read it in the paper: doi.org/10.1016/j.omta.2026.201835.

Versions

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Version 2, 28 September 2026

  • Authors: added Yuishin Izumi (0000-0002-3048-9041); Haruhisa Inoue (0000-0003-4736-9537); removed Yuishin Izumi; Haruhisa Inoue
  • Funding: added Japan Agency for Medical Research and Development

Version 1, 27 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 24 authors, 10 keywords, 43 references, 1 RRID.

Cite

This paper

Imamura, K., Yoshioka, S., Kamizato, K., Oki, R., Hioki, H., Tsukita, K., Inoue, I., Shimizu, R., Takiguchi, M., Sakasai, T., Okusa, A., Shibukawa, R., Niki, T., Sakurai, S., Okunomiya, T., Kondo, T., Kato, T., Miyanohara, A., Kakinohana, M., . . . Inoue, H. (2026). Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models. Molecular therapy. Advances, 34(4), 201835. https://doi.org/10.1016/j.omta.2026.201835

BibTeX

@article{imamura2026base,
author = {Imamura, Keiko and Yoshioka, Shin and Kamizato, Kota and Oki, Ryosuke and Hioki, Hiroyuki and Tsukita, Kayoko and Inoue, Ikuyo and Shimizu, Rina and Takiguchi, Mako and Sakasai, Tomoki and Okusa, Aya and Shibukawa, Ran and Niki, Takeshi and Sakurai, Satoko and Okunomiya, Taro and Kondo, Takayuki and Kato, Tomohisa and Miyanohara, Atsushi and Kakinohana, Manabu and Yamamoto, Takuya and Marsala, Martin and Nishida, Keiji and Izumi, Yuishin and Inoue, Haruhisa},
title = {{Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models}},
journal = {Molecular therapy. Advances},
year = {2026},
month = aug,
volume = {34},
number = {4},
pages = {201835},
publisher = {American Society of Gene \& Cell Therapy},
issn = {3117-387X},
doi = {10.1016/j.omta.2026.201835},
url = {https://doi.org/10.1016/j.omta.2026.201835},
pmid = {42740795},
pmcid = {PMC13571996}
}

RIS

TY - JOUR
AU - Imamura, Keiko
AU - Yoshioka, Shin
AU - Kamizato, Kota
AU - Oki, Ryosuke
AU - Hioki, Hiroyuki
AU - Tsukita, Kayoko
AU - Inoue, Ikuyo
AU - Shimizu, Rina
AU - Takiguchi, Mako
AU - Sakasai, Tomoki
AU - Okusa, Aya
AU - Shibukawa, Ran
AU - Niki, Takeshi
AU - Sakurai, Satoko
AU - Okunomiya, Taro
AU - Kondo, Takayuki
AU - Kato, Tomohisa
AU - Miyanohara, Atsushi
AU - Kakinohana, Manabu
AU - Yamamoto, Takuya
AU - Marsala, Martin
AU - Nishida, Keiji
AU - Izumi, Yuishin
AU - Inoue, Haruhisa
TI - Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models
T2 - Molecular therapy. Advances
J2 - Mol Ther Adv
PY - 2026
DA - 2026/08/21
VL - 34
IS - 4
SP - 201835
SN - 3117-387X
PB - American Society of Gene & Cell Therapy
DO - 10.1016/j.omta.2026.201835
UR - https://doi.org/10.1016/j.omta.2026.201835
LA - en
ER -

CSL-JSON

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