M. Kakinohana

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...