Dr. Sean J. Miller is a translational neuroscientist focused on developing human-relevant experimental models to uncover disease mechanisms and accelerate therapeutic discovery for neurological and neurodegenerative disorders. His research integrates human induced pluripotent stem cell–derived neural and retinal organoids, primary human tissues, single-cell and spatial transcriptomics, proteomics, advanced microscopy, and functional electrophysiology to define how disease-associated molecular and cellular changes translate into neural dysfunction. By combining patient-derived biology with mechanistic experimentation and therapeutic perturbation, his laboratory aims to bridge the gap between human disease discovery and preclinical drug development. The overarching goal of his research is to identify actionable pathways, validate therapeutic targets in human systems, and develop interventions with a clear path toward clinical translation.