Restoring SCN1A-Mediated Neuronal Excitability Through Cell-Selective Gene Regulation in Clinical Dravet Syndrome
- Demonstrating durable SCN1A upregulation using AAV9-based gene regulation in clinical development
- Translating cell-selective targeting of inhibitory neurons into disease-modifying potential in Dravet syndrome
- Optimizing precision gene control strategies to improve safety and efficacy in CNS ion channel disorders