NeurosciencebioRxivPreprint
CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability
Laura Sichlinger, Molly B. Reilly, Sakshi Arora…·Elizabeth A. Heller
This paper demonstrates that CRISPR-mediated transcriptional activation can upregulate SYNGAP1 in a mouse model of SRID and human neurons, rescuing deficits without targeting mutations.