CRISPR-Based Precision Epigenetic Editing of Methylation Patterns in Neural Stem Cells Derived from Patients with Rett Syndrome
Keywords:
Rett syndrome, CRISPR-dCas9, DNA methylation, neural stem cells, epigenetic editing, MECP2Abstract
Rett Syndrome (RTT) is a neurodevelopmental disorder predominantly affecting females and is primarily caused by mutations in the MECP2 gene. This study investigates the application of CRISPR-dCas9 tools fused to epigenetic modifiers to target and reverse abnormal methylation in neural stem cells (NSCs) derived from RTT patients. Using site-specific demethylation of MECP2 promoter regions, we observed restored gene expression and partial rescue of neurogenic function in vitro. The study provides a proof-of-concept for precise epigenetic editing in disease modeling and potential therapeutic intervention.
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