Author

Kayoko Tsukita

3 papers indexed here

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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

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

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