Developing drugs using pluripotent stem cells and genomics data

Induced pluripotent stem cells (iPSCs), formed from cell samples of patients with known disease by inserting four specific transcription factor genes into the cell chromatin, can be used to identify compounds that directly reverse the disease showing up in cells grown out (differentiated) to the mature state e.g. neurones or heart muscle etc. cell types. Human genetics data can also identify potential genes or gene pathways that may cause disease and the use of reverse pharmacology can be used to show in isogenic iPSCs (different genetically by editing out the one targeted gene selected) that disease type can be reversed by drugs targeting these genes. These powerful new approaches are displaying a whole new arsenal of drugs for disease.