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,...