Author

Satoko Sakurai

2 papers indexed here

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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

MYCN reprograms postnatal cells toward embryonic-like transcriptional states and drives tumorigenesis.

The embryonic-like features of neuroblastoma are commonly explained by a developmental arrest model, in which neural crest-derived progenitors undergo transformation after failing to complete normal sympathoadrenal differentiation. Whether such features can instead be actively imposed on postnatal cells during tumor de...

Y. Nakatsukasa, Michitada Hirano, Yosuke Yamada et al. · Biochemical and Biophysical... · 0 citations