Developing drugs using pluripotent stem cells and genomics data

Lead researcher

Jin Zhang

Main finding

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.


Centre for Cancer Research

Research group

Stem cell immunotherapies


Hu Li, Alan Trounson, Joseph C Wu, Paul Nioi

Journal and article title

Most surprising

The power of pluripotent stem cells to provide new insights and data on the genetic cause of disease and identification of potential drugs to treat complex disease.

Future implications

Provides many new opportunities to stabilise or cure disease.

Disease/health impact

Multiple disease states

Other points of interest

Gene editing and iPSC biology are the new frontiers in medical research.