Author

Aya Okusa

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#gene editing Open access Sep 2026

Mutation-specific correction of SOD1 in familial ALS using prime and base editing in human induced pluripotent stem cells

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive motor neuron degeneration and respiratory failure. Approximately 10% of ALS cases are familial, with mutations in SOD1 representing a major genetic cause. Preservation of physiological SOD1 function may be i...

K. Imamura, Kayoko Tsukita, Shin Yoshioka et al. · BMC Medical Genomics · 0 citations

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