CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability.

CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability.
Sichlinger L, Reilly MB, Arora S, Zhang S, Marotta N, Rodríguez-Acevedo KL, Hooks M, Czarnecki KS, Winter JJ, Waxman EA, Dungan LV, Hong I, Araki Y, Johnson R, Huganir RL, Pavani G, French DL, Davidson BL, Prosser BL, Heller EA.
bioRxiv. 2025 Oct 29:2025.10.28.685100. doi: 10.1101/2025.10.28.685100. PMID: 41279390. [Preprint]