CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder
Serena Tamura, Andrew D. Nelson, Perry W.E. Spratt, Elizabeth C. Hamada, Xujia Zhou, Henry Kyoung, Zizheng Li, Coline Arnould, Vladyslav Barskyi, Beniamin Krupkin, Kiana Young, Jingjing Zhao, Stephanie S. Holden, Atehsa Sahagun, Caroline M. Keeshen, , Kevin J. Bender
This study shows that CRISPR activation can rescue electrophysiological deficits in SCN2A haploinsufficiency by increasing the expression of the remaining functional allele in mice and human neurons.
Nature · Sep 17, 2025Peer-reviewedNeuroscience