Developmental and Epileptic encephalopathy (DEE) refers to the severe drug-resistant epilepsy disorders that cause developmental delays or the loss of developmental skills. The dynamin-1 gene (DNM1) causes a particularly severe form of DEE. Currently, there is no treatment for DNM1-related DEE; however, Dr. Scott Harper, principal investigator at Nationwide Children’s Hospital’s Gene Therapy Center, developed an RNAi therapy targeting DNM1 that will decrease the expression of pathogenic variants in the DNM1 gene and can be applied to any dominant DNM1 variant. The approach knocks down either of two mutually-exclusive RNA splice forms. The therapy will target either of the isoforms to significantly reduce the symptoms of DEE in patients.