Stanford researchers have developed a new gene editing approach that enables red blood cell-specific gene expression for the treatment of enzyme deficiencies.
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.
Polycythemia vera is a rare blood cancer characterized by the hyperproliferation of red blood cells, leading to coagulation events like strokes and heart attacks.