Stem cells can self-renew and give rise to differentiated progeny. For these characteristics stem cells have attracted a lot of attention as they offer unique possibilities to study the development of specific cell types, and for therapeutic purposes. Since it is now possible to derive induced pluripotent stem cells (iPSCs) from somatic cells the potential use of stem cells has dramatically increased. The research in our lab is focused on the use of iPS cells for therapeutic purposes. To this end we not only study the differentiation of pluripotent stem cells into different lineages in vitro but also transplant these cells in vivo.
For the differentiation of cells we look into long non-coding RNAs (lncRNAs). There are many transcribed regions in the genome but of the majority the exact function is elusive. We and others have hypothesized that these lncRNAs are extremely important for proper differentiation of stem cells into specific cell types along the course of development. One of the lncRNA, we are interested in, appears to be important for neural differentiation. To identify the function of these genes we genetically manipulate ES cells, and differentiate these into neural cell types.
To study blood disorders we are building iPSC based blood models. These models are used to identify mechanisms underlying pathogenic phenotypes of rare blood disorders. In addition, they allow functional analysis of iPSC derived progeny, either cells from healthy individuals or from diseased cells in which the causal mutation has been repaired.
Furthermore we run the LUMC iPSC core facility together with the group of Prof. dr. Christine Mummery. In this facility we have generated numerous control and disease-specific iPSC lines from different tissue origin (blood, urine, skin) using integrating (lentiviral vectors) as well as integration-free (Sendai, plasmid) methods.