?-thalassemia is a devastating blood disorder caused by mutations in the HBB gene encoding ?-globin, where treatment involves lifelong, costly management of the resulting lack of hemoglobin and hemolytic anemia.
Researchers at Stanford have developed gene editing methods for modifying hematopoietic stem and progenitor cells (HSPCs) to express truncated forms of the erythropoietin receptor (tEPOR).