Gene Therapy as a Curative Option for β-Thalassemia

With an estimated global prevalence of 288,000 cases, β-thalassemia is one of the most common genetic diseases in the world, and every year another 60,000 infants are born with the disease. Of these patients, 60 to 80% have a severe form of the disease and require regular red-cell transfusions and iron chelation for proper treatment of disease manifestations. In the United States and the European Union, the prevalence is estimated to be 15,000, with approximately 1500 infants born each year with the disease. However, in North Africa, the Middle East, and Asia, β-thalassemia is more prevalent. In many of these . . .