FMR1 Gene Therapy Reduced Seizures and Gamma EEG Power in Fragile X Mice
TL;DR: A 2026 mouse study in Gene Therapy used an adeno-associated virus 9 (AAV9) vector to deliver human FMR1 and reduced sound-triggered seizures, excessive digging, and gamma-frequency electroencephalography (EEG) power in selected dose and route groups. The outcomes came from separate experiments, and high-dose intravenous treatment without immunosuppression caused excess deaths. Key Findings Adolescent seizure …
