CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder
Serena Tamura, Andrew D. Nelson, Perry W.E. Spratt…·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.