CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability
Laura Sichlinger, Molly B. Reilly, Sakshi Arora, Shuo Zhang, Nicolas Marotta, Kiara L. Rodríguez-Acevedo, Marisol Hooks, Kyle S. Czarnecki, Julia J. Winter, Elisa A. Waxman, Lea V. Dungan, Ingie Hong, Yoichi Araki, Richard C. Johnson, Richard L. Huganir, , 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.
bioRxiv · Oct 28, 2025 · PreprintNeuroscience