Potential application of embryonic stem cells in Parkinson’s disease: drug screening and cell therapy

Abstract

Embryonic stem (ES) cells are genetically normal, continuous cell lines that can give rise to a variety of somatic cells in culture. These include the midbrain dopaminergic neurons, a major cell type lost in Parkinson’s disease. With the promising outcome of mesencephalic fetal transplantation in some Parkinson’s disease patients, the establishment of human ES cells has sparked much attention in both the scientific and general community regarding their potential as an alternative to aborted fetal tissue for cell replacement therapies. There is also great interest in developing the ES cell system as a platform for pharmaceutical and toxicological screening. Progress has been made in developing protocols for dopaminergic neuronal specification in ES cell development. Research to define the criteria for the ‘right’ category of therapeutic dopaminergic cells is underway. However, the promise of human ES cells rests largely on our ability to expand stem cells without genetic and epigenetic compromise, and to direct stem cell differentiation with absolute phenotypic fidelity. The delivery of these goals will require a much better understanding of the control of ES cell self-renewal, proliferation and the commitment of differentiation.