Human induced pluripotent stem cells (hiPSCs) have evolved as an important human in vitro model for biomedical research. This model allows to differentiate specific human cell types at scale to study human disease mechanisms in a cell culture dish. The versatility of this model is further enhanced by combination with Clustered regulatory interspaced short palindromic repeats (CRISPR)-associated nuclease (CAS)-mediated genetic engineering to study gene effects and disease mechanisms in isogenic hiPSC lines. Therefore, CP1 cooperates with ProBone projects to utilize early-onset low BMD patient-derived primary cells, reprogramme hiPSC and establish isogenic controls by CRISPR/Cas9. Differentiation protocols into bone cell types will be established and 2D and 3D microphysiological - and organoid bone models will be generated. In essence, CP1 will establish human in vitro disease models to advance understanding of early-onset low BMD mechanisms.

Institute of Experimental Pharmacology and Toxicology