CRISPR-mediated Stxbp1 gene activation restores Munc18-1 levels in Stxbp1-haploinsufficient mice
Tetsushi Yamagata, Ryu Ikebe, Kenta Kobayashi, Masanari Saito, Yurina Hibi, Kazuhiro Yamakawa, Toshimitsu Suzuki
This study uses CRISPR-ON to increase Stxbp1 expression in a mouse model, restoring Munc18-1 protein levels but not fully reversing disease symptoms.
bioRxiv · Jun 9, 2026 · PreprintNeuroscience