Throughout her career, Kathleen Millen has leveraged the power and strengths of both mouse and human genetics, with embryological analysis in mice and other model vertebrates, to identify the molecular and cellular events that regulate brain development and contribute to human neurodevelopmental disorders. She has long focused on congenital structural malformations of the cerebellum and, more recently, cerebral cortical malformations. Millen has made foundational discoveries regarding the genetic control of early brain development and identified genes and processes underlying several birth defects of the human cerebellum and is currently also working on developing novel therapeutic strategies for intractable pediatric epilepsy.

Together with Kimberly Aldinger, an expert in human molecular genetics and neural development, Millen has initiated a project to define the important relationship between molecular cell types in the cerebellum and autism pathophysiology. Elucidating the molecular role of the cerebellum in autism will not only close an important knowledge gap and guide future autism research, but is also likely to provide new avenues to target for cellular therapies.