Very recently, it has been found that the activation of four specific “reprogramming” genes in mature cells taken from individual patients (from the skin, for example), can revert the cells into embryonic stem cell-like cells. The resultant so-called induced pluripotent stem (iPS) cells can then be turned into the cell types or organs needed. Patients with a range of diseases could potentially be cured by their own cells, with no need to take the immunosuppressant drugs that are necessary after conventional transplants.
Aims and Relevance
The current iPS cell inducing technology is not yet patient friendly. The four reprogramming genes are introduced into adult cells by viruses that randomly integrate into the cell’s DNA. This could lead to mutations in important genes and cause additional diseases. It would be preferable to replace the reprogramming genes with growth factors or other small molecules that can be easily added to the cell culture medium. We are currently conducting large-scale screens to identify appropriate small molecules for this task. We are also developing protocols to revert hematological cells into iPS cells; these studies will lay the foundation for revolutionizing the treatment of many patients with otherwise fatal hematologic diseases and as a by-product, allow the development of new and powerful systems for studying the development and better treatent of leukemia.