Therapeutic uses of iPS cells and as models in pathology

The fifth lecture focused on the application of iPS cells as a therapeutic tool and as a model in pathology. A first consideration concerns the possible dangers of these cells in the clinic. Although iPS cells avoid the technical and ethical drawbacks of cloning, the use of vectors of retroviral origin to introduce OKSM transcription factors has been associated with risks of random insertion into the genome. Different strategies for introduction, or even chemical induction of transcription factor expression, have been discussed. Moreover, the use of a potential oncogene - MYC - in Yamanaka's original cocktail was replaced by that of other factors, thus considerably reducing the risks of oncogenesis. Nevertheless, the inefficiency of the iPS cell production procedure and the persistent difficulties in differentiating these cells into specific pathways make therapeutic prospects in humans uncertain. Conversely, the use of iPS cells to explore cardiac, hematopoietic, neurodegenerative or other diseases is booming, with great hope for a better understanding of the molecular basis of certain disorders and treatment prospects. Indeed, the derivation of iPS cells from patients or healthy individuals enables the screening of drugs in a "personalized" way, and represents a booming field. This last lecture concluded with a discussion of the use of adult stem cells or trans-differentiated cells for therapeutic purposes, with the example of treatments in development using trans-differentiated cells created in situ, following a heart attack.