IPEX syndrome is a severe autoimmune disease with limited treatment options caused by mutations in the forkhead box protein 3 (FOXP3) gene, which plays a critical role in immune regulation.
Polycythemia vera is a rare blood cancer characterized by the hyperproliferation of red blood cells, leading to coagulation events like strokes and heart attacks.
Researchers at Stanford and the Chan Zuckerberg Biohub have developed a methodology to monitor cell expansion and differentiation following targeted genomic modification.