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
Stem cell immunotherapies
Journal and article title
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.
Provides many new opportunities to stabilise or cure disease.
Multiple disease states
Other points of interest
Gene editing and iPSC biology are the new frontiers in medical research.