Preclinical Development of an AAV-Based Gene Therapy for FRRS1L Epileptic Encephalopathy
2:25 pm - Thursday 26th March 2026
- Gaining insight into cutting-edge AAV9-mediated FRRS1L gene therapy development for a devastating epileptic encephalopathy
- Learning how dose-dependent efficacy and molecular restoration in knockout models support the therapeutic potential of FRRS1L replacement
- Discovering how these findings inform IND-enabling studies and the broader advancement of gene therapy for neurodevelopmental disorders
