SYNGAP1-related disorder: pathophysiology, epilepsy, cognitive and behavioral phenotypes, and precision therapeutic approaches.
A rapidly advancing precision-therapy pipeline-including antisense oligonucleotides to upregulate the intact allele, AAV-based gene replacement, CRISPR-mediated transcriptional activation, epigenetic modulators, and rational pathway-targeted small molecules-offers realistic prospects for disease modification.
Debopam Samanta
· Epilepsy & Behavior · 2 citations