Reverse translational research takes a bedside to bench approach, using sophisticated basic research to explain the biological mechanisms behind observed clinical data. For transporters, which play a role in human disease and drug response, this approach offers a distinct advantage over the typical translational research, which often falters due to inadequate in vitro and preclinical animal models. Research on ABCG2, which encodes the Breast Cancer Resistance Protein (BCRP), has benefited immensely from a reverse translational approach due to its broad implications to disease susceptibility and both therapeutic and adverse drug response. In this review, we describe the success of reverse translational research for ABCG2 and opportunities for further studies. This article is protected by copyright. All rights reserved.