Research in neuroscience is poised to play an essential role in efforts underlying Columbia’s precision medicine-based approaches to the diagnosis and treatment of neuropsychiatric disease . There is now compelling evidence that rare but highly penetrant mutations in a large number of single genes may account for a significant fraction of cases of brain diseases, including autism spectrum disorder, schizophrenia, bipolar disorder and epilepsy. With recent and future advances in genome sequencing, the number of genes implicated in such diseases is rising at an ever-increasing rate.
Basic neuroscience research at the molecular, cellular and systems levels will play a critical role in elucidating the mechanisms by which these gene mutations lead to disease. Such efforts may help in reclassifying disease phenotypes based on molecular mechanisms, rather than relying on exclusively behavioral-clinical diagnoses. Importantly, such research will provide important information about new targets for therapeutic interventions that may lead to novel, mechanism-based precision treatments for these devastating disorders.