While there is limited data on sex-dependent differences of AAV-gene therapy in the CNS, one recent study demonstrated sex-specific responses to intracerebroventricularly delivered AAV9 in a mouse model of Dravet syndrome, a debilitating seizure disorder caused by mutations in the α subunit of NaV1.1 channels (SCN1A) [80]. This evidence concerns the gene SCN1A and encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy.