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September 10, 2025Open Access

Hypoimmunogenic human motor neurons induced from iPSCs in vivo substantially ameliorate ALS disease in large animal models

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Authors

NZNa ZhangYYYang YangMCMengqi Chen

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Overview

Stem cell therapy shows promise, significantly improving motor function in ALS models via genetic editing.

Key Points

  • Transplanted hypoimmunogenic motor neurons substantially ameliorated ALS symptoms in large animal models.
  • Doxycycline-inducible transcription factors enabled these iPSC-derived motor neurons to functionally integrate into host circuits.
  • Multiplex genetic-editing was employed to modify immunomodulatory genes for optimal cell integration and survival.
  • Long-term survival without immunosuppression in ALS models points to a breakthrough in stem cell therapy for neurodegenerative diseases.

Cite This Study

Zhang et al. (2025) studied this question.

synapsesocial.com/papers/68c194029b7b07f3a06188e3https://doi.org/10.1101/2025.09.03.673895
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