Open access
Aug 2026
Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models
In hereditary motor neuron diseases (MNDs), including forms of amyotrophic lateral sclerosis (ALS) caused by single-nucleotide variants, effective therapeutic strategies need to address both gain- and loss-of-function mechanisms. Genome editing-based gene therapy represents a promising approach for simultaneously targe...
K. Imamura, Shin Yoshioka, Kota Kamizato et al.
· Molecular therapy. Advances · 0 citations