Introduction: Blood cell transfusion plays a vital role in modern medicineāsupporting surgery, obstetrics, trauma care, and cancer chemotherapy. In the US alone, more than 12 million red-cell units are consumed annually.
?-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 a potentially curative treatment strategy for alpha-thalassemia, one of the most common autosomal recessive disorders in the world involving the genes HBA1 and/or HBA2.
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).
Polycythemia vera is a rare blood cancer characterized by the hyperproliferation of red blood cells, leading to coagulation events like strokes and heart attacks.
Despite their rapidly expanding therapeutic potential, human pluripotent stem cell (hPSC)-derived cell therapies continue to have serious safety risks.