A possible therapeutic approach to curing PD is to generate immune compatible dopaminergic neurons by reprogramming somatic cells (e.g., skin fibroblasts or blood cells) from PD patients and then differentiating them into dopaminergic neurons. To do this in a clinically compliant manner, it is important to show that scalable methods of GMP-compatible cell culture exist, and that iPSC lines are overall similar to hESCs so that the body of existing literature on hESCs can be utilized. It is equally important to show that iPSC lines can be obtained from disease models of PD, and that these lines can be propagated and differentiated appropriately, and used for comparative analysis. Finally, it is also important to show that a stage(s) where the effect of a mutation can be assessed and that the mutation can be corrected or a mechanism be identified.
