Pluripotent stem cells for study of development, disease and cell therapy
Embryonic stem cells are pluripotent stem cells (PSCs) obtained by extraction and culture of the inner cell mass from a blastocyst—an early stage of development following fertilisation of a mammalian egg. These cells can be cultured in vitro and, in principle, used as a source of any cell type in the mammalian organism. It is now possible to obtain patient-derived 'induced' pluripotent stem cells (iPSCs) from skin biopsies or blood samples.
PSCs can be encouraged to differentiate in culture under the influence of specific growth factors or small molecule agents to produce a wide variety of cell types. However, there is much work to be done to define protocols to enable precise control of differentiation to obtain all cell types. D4 research is engineering genetic reporters into mouse and human PSCs to track the progress of differentiation towards particular cell types.
The opportunity to obtain PSCs offers the potential to study disease in humans, comparing differentiated cells from individuals to identify signalling pathways that influence the course of disease. D4 researchers are investigating using this technology for the study of neurological and neurodegenerative diseases—in particular Parkinson's and Alzheimer's diseases. We also have interests in modelling the function of cardiomyocytes, kidney cells and peripheral neurons that sense pain.
PSCs are also being investigated as cell sources for direct cell therapy by transplantation, and in regenerative medicine where the aim is to replace damaged tissue. D4 researchers are collaborating with Australian and international groups in the study of cell transplants into the brain for the treatment of neurodegenerative diseases.