PV interneuron-targeted CRISPRa rescue of SCN1A haploinsufficiency in Dravet syndrome
Perry W.E. Spratt, Nicholas F. Trojanowski, Rajani M. George…·Kathryn C. Allaway
This study uses CRISPR activation to upregulate SCN1A in PV interneurons, improving survival and reducing seizures in Dravet syndrome models.