Figure 4.

Current and Future Development of iPSC-based technology in BMP rare diseases. Since the inception of the first iPSC-based cell models, significant progress has been made in developing more intricate and robust systems that accurately replicate diseased tissues and organs. Of particular interest in monogenic diseases is the utilization of gene editing tools such as CRISPR/Cas9 to create isogenic cell lines. While the first culturing approaches opted for 2 dimensional methods, more complex three dimensional culture conditions, such as embryoid bodies (EBs) and organoids, are emerging. This can be combined with co-culture of different cell types differentiated from the same parental iPSC line. Culturing those cells under conditions that expose them to microenvironmental cues, such as hypoxia, biomechanics, and inflammation, allows for effective and complex disease modelling. These systems have proven to be valuable platforms for drug screening and in-depth mechanistic investigations. Further advances, such as construction of complex cellular grafts or therapeutic extracellular vesicles (EVs) derived from iPSCs, hold the potential to revolutionize tissue engineering and iPSC-based cell therapies, presenting therapeutic alternatives for patients affected by rare BMP diseases.