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Molecular Therapy Advances

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

By Keiko Imamura, Shin Yoshioka, Kota Kamizato, Show 20 more authorsHaruhisa Inoue
Experiments in animalsInterventions
Paper

The paper

Keiko Imamura, Shin Yoshioka, Kota Kamizato, Show 20 more authorsHaruhisa Inoue

Kyoto University

Molecular Therapy Advances, 21 Aug 2026

doi.org/10.1016/j.omta.2026.201835PubMed 42740795

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