Author

Tomoki Sakasai

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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

Open access Aug 2026

Generation of mutant human SOD1 knock-in mouse lines at the Rosa26 locus as a platform for developing genome-editing therapies for amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by the degeneration of upper and lower motor neurons, resulting in progressive paralysis and death within a few years of symptom onset. Although current treatments modestly slow the disease progression, effective disease-modify...

T. Okunomiya, Tomoki Sakasai, Kayoko Tsukita et al. · Experimental animals · 0 citations