Stem Cell Engineering

Somatic cells can be reprogrammed to a pluripotent embryonic stem cell-like state (induced pluripotent stem (iPS) cells) by combinations of the transcription factors OCT4, SOX2, NANOG, LIN28, KLF4 and cMYC. We were able to demonstrate, that mouse and human neural stem cells, that express SOX2, KLF4 and cMYC, can be reprogrammed to iPS cells by expression of OCT4. We have comparatively evaluated the hematopoietic and erythroid as well as neural differentiation potential of human cord blood and neural stem cell derived iPS cells. Patient-derived induced pluripotent stem cells and their differentiation derivatives hold great promise as novel in vitro model systems to study human pathogenesis. We are currently focusing to model neural (Frontotemporal Dementia) and skeletal muscle (Duchenne, LGMD) diseases with human iPS cells and genome editing.