The production of cellular therapies requires the optimization of four steps: first, isolating and culturing cells that can be readily obtained from a patient in a non-invasive fashion. Second, the reprogramming of these cells into a pluripotent state. Third, the directed differentiation of those patient-specific pluripotent cells into the cell type relevant to their disease. And, fourth, techniques for repairing any intrinsic disease-causing genetic defects and transplantation of the repaired, differentiated cells into the patient. Notably, these disease-relevant patient cells can also be used for in vitro disease modeling which may yield new insights into disease mechanisms and drug discovery.
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