Pediatric epileptic encephalopathy causes severe seizure and significant developmental delays or the loss of developmental skills. Currently, treatment is limited to symptomatic treatment through the primary use of antiepileptic drugs. Researchers at Nationwide Children’s Hospital developed a gene therapy for the disorder using six different single stranded Adeno-Associated Virus vectors serotype 9 (AAV9) to combat mutations in the SLC6A1 gene that causes neurological disorders like pediatric epileptic encephalopathy. The AAV9 vectors will deliver a wildtype copy of SLC6A1 to address haploinsufficiency resulting from mutations in one copy of the SLC6A1 gene. The gene therapy will be delivered through the cerebrospinal fluid (CSF) via injection methods like intracerebroventricular, cisternal or lumbar intrathecal injection. Current treatment for pediatric epileptic encephalopathy does not address the underlying genetic defect nor does it slow or prevent the progression of the disease, this new innovative gene therapy will target the genetic defect in hopes of slowing or preventing its progress.