The large sphingolipid accumulation and microglial dysfunction during larval stages shows the potential to use the gba1 mutant zebrafish as a tool for phenotypic drug discovery to identify new disease modifying therapies for neuronopathic GD and to aid in the identification of novel PD toxins that may act synergistically in conjunction with gba1+/−. Here, GBA1 is linked to Parkinson disease.