In the first year of the CIRM Early translational research award, we established a bank of 51 cell lines derived from skin cells of patients with Parkinson’s disease that carry specific mutations in known genes that cause PD as well as sporadic PD patients. We also recruited matched healthy individuals that serve as controls.
In a next step, we reprogrammed (‘rejunivated’) 17 samples of skin cells to derive pluripotent stem cells (iPSC) that closely resemble human embryonic stem cells characterized by biochemical and molecular techniques. We also optimize this process by introducing factors the will be removed after successful reprogramming.
We have now built a foundation for the next milestones and made already progress on the differentiation into authentic dopamine producing cells, and we have developed assays to assess the Parkinson’s disease-specific pathological phenotype of the dopamine neurons.