In addition to the value of new models suggested by genetic analysis, there are several opportunities to exploit advances in diagnostic and therapeutic genetic approaches. There are now multiple examples of strategies one could use to develop gene therapy employing viral vectors to treat focal and generalized epilepsies in animal models in which a missense variant or truncating mutation has modified the function of a target gene or reduced the gene dose.42 Other innovative uses of gene therapy include introduction of potassium channels that could reduce excitability, as well as engineering cells to release neuroactive molecules that can counteract excessive excitability.43,44 As more animal models are developed for different genes, there will be opportunities to test fundamental approaches that supplement underexpressed alleles or proteins with reduced function, as well as editing gene approaches to correct identified defects. These strategies will require demonstration of utility in animals with measurable defects, and the results will speak to the important question in epilepsy around whether symptoms are driven by the genetic defect, are a feature of maladaptive compensation, or reflect some combination of both. That is, there is a need for proof-of-concept data for oligonucleotide and antisense therapies for application in the treatment of genetically defined monogenic epilepsies, as well as data on effectiveness of the timing of treatment in the context of the development of a seizure focus. Advances are needed in genetic therapy using virus delivery vectors that are already approved for other payloads and access both brain and spinal cord following intrathecal administration. The rare genetic epilepsies might provide a test case for intervention, which can be evaluated in iPSC-based models in vitro, organoids derived from iPSC cells, and animal models now.