Open access
Aug 2026
Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models
Findings support the therapeutic potential of base editing for hereditary MNDs by identifying the optimal adenine base editor and evaluating the selected editor by subpial delivery of adeno-associated virus (AAV) vectors to the spinal cord.
K. Imamura, Shin Yoshioka, Kota Kamizato et al.
· Molecular therapy. Advances · 0 citations