Preprint on CRISPRa approach to rescue SCN2A haploinsufficiency posted

Autism-associated variants in the gene SCN2A commonly impair gene function. This leads to haploinsufficiency, where a single functional copy cannot compensate for the loss of its partner. Here, Serena Tamura, a recently-graduated student in the Ahituv lab at UCSF, worked with us to test whether CRISRP activators can rescue SCN2A haploinsufficiency. See here for more info: 

Also, you can check out this twitter thread where we provide a bit more commentary.