Exacerbation of a Subset of Behavioral Phenotypes by Early Treatment with AAV FOXG1 Gene Replacement Therapy in a Mouse Model of FOXG1 Syndrome
Christina L Torturo, Kimberly Kerker, Jodi E. Gresack, Christopher DeGrave, Ailing Du, Dan Wang, Guangping Gao, Scott Reich, Sylvie Ramboz, Dinah W.Y. Sah
This study shows that early AAV FOXG1 gene therapy in female mice worsens motor behaviors, while it benefits males, highlighting gender-specific risks in potential human treatments.
bioRxiv · Oct 22, 2025 · PreprintNeuroscience