The Cell Programming Core Facility exploits the scientific know-how of the Institute of Reconstructive Neurobiology in cellular (re)programming, genome engineering and stem cell differentiation to support the establishment of cellular disease models on the basis of patient-specific stem cells. We expect that models, which are based on authentic human cells will provide new insights into the molecular pathogenesis of diseases and promote the development of pharmaceutical and cell-based therapies.
For many disorders, a major challenge in studying their molecular pathomechanisms is the limited experimental access to disease-affected human tissue or cells. Since in many cases the molecular disease initiation occurs years or decades before patients develop symptoms, biopsy or autopsy specimens can only reflect the final phase of the disease. Novel cell (re)programming approaches such as the generation of human induced pluripotent stem (iPS) cells from somatic cells such as fibroblasts or peripheral blood provide access to virtually unlimited numbers of patient-specific stem cells for the study of disease-relevant pathomechanisms. In addition, the successful identification of early pathological alterations should to pave the way to uncover novel drug targets for therapeutic intervention.