Gene therapy for β-thalassemia syndromes

-thalassemias are among the most common inherited single-gene defects worldwide with a global birthrate incidence of 40.000/year, mainly in the Mediterranean, Middle East and Southern Asia countries. In Italy alone, there are over 5000 patients and about 1.5 million carriers of the trait. This autosomal recessive disorders results from the absence or insufficient production of hemoglobin -chains that determinates a degree of imbalance in the α-and -globin protein, which is directly linked to the clinical severity of the disease. The genetic defect hinders precursor maturation and results in ineffective erythropoiesis and hemolytic anemia, which usually become evident a year after birth and, in the severe form of the disease, is fatal within the first decade of life...