Integrating stem cell-based experiments in clinical research

Abstract

With the seminal discovery of somatic cell reprogramming with defined genetic factors, it is now a routine laboratory procedure to reprogram somatic cells to generate patient-specific induced pluripotent stem cells (iPSCs) [1] Patient-specific iPSCs can be differentiated to generate mature neurons as well as three-dimensional brain organoids that show appropriate functional activity in electrophysiological studies [2,3]. However, there is a significant gap in the thoughtful incorporation of patient-derived neuronal cells in clinical studies addressing disease risk.