A schematic diagram of a possible approach to using a stem cell-based system in a drug discovery campaign. The central idea is that the cells used for screening, efficacy and safety testing would be patient-derived. Also, lines of cells, potentially prepared from many patients, would be used for efficacy and safety testing in vitro prior to testing on those patients in vivo. It is hoped that this will increase the probability of clinical success.
Rubin and Haston BMC Biology 2011 9:42 doi:10.1186/1741-7007-9-42