
Dr. Zervas utilized a naturally occurring mouse model of Niemann-Pick Disease Type C (NPC) and a feline model of NPC to understand how gangliosides, unesterified cholesterol, and disease pathology can be ameliorated using the ganglioside synthesis inhibitor NB-DNJ, which was the discovery work that directly led to the first approved therapy (Miglustat) to treat human NPC (outside the US). However, in 2024 a therapy for NPC (Miplyffa) was approved in combination with Miglustat, to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Dr. Zervas has a background in mouse genetics and established Genetic Inducible Fate Mapping (GIFM) to label and track neuronal lineages defined by gene expression. He built upon this work to combine GIFM and conditional gene deletion to simultaneously mark and mutate specific lineages at discrete embryonic time points. This combined approach was used to establish a novel mouse model of Tuberous Sclerosis and to elucidate molecular programming of VTA dopamine neurons.