Molecular Cell Biology 2

Therapeutic Cell Differentiation (5.03.02)

Principal investigators

Prof. R. C. Hoeben, Prof. Dr. M.J.T.H. Goumans, Dr. A. Zaldumbide, Dr. M.F.V. Gonçalves, Dr. H.M.M. Mikkers, Dr. M.E. Ressing

Aim and focus   

The mission of this program is to develop stem cell-based regenerative medicine approaches for genetic and acquired diseases, such as heart failure, pulmonary hypertension, type I diabetes, primary immune deficiencies, and muscular dystrophy. We use genetic modification for correcting genetic defects and protecting grafted cells from the effects of the host’s immune system. The research is focused on understanding the molecular and biochemical cues such as TGFb signaling that govern cell differentiation and behavior. This knowledge is employed for steering the differentiation of cells in vitro and in vivo. Furthermore, using e.g. growth-factor cocktails, exosomes, and transcription factors, we explore the capacity of stem cells to home to sites of injury and repair the tissue from within, or to migrate to tumors for the delivery of therapeutic agents, such as oncolytic viruses.

For preclinical evaluation of our strategies in animal models for human disease we have engaged clinical departments both nationally and internationally. The group’s genetic modification platform is employed to transfer reporters to facilitate tracking and tracing of stem cells and their progeny, linking this group’s activities to the Biomedical Imaging profiling area. The genetic modification technology is made available to LUMC researchers via the Viral Vector Facility. In addition, our group employs induced pluripotent stem cells (iPSCs), and in collaboration with the department of Embryology and Anatomy, a core facility dedicated to the generation of iPSCs is run.