Thalassemia and Cell Based Gene Therapy (Zynteglo)

Abstract

Thalassemia is a group of inherited blood disorders characterized by decreased or absent production of one or more of the globin chains that make up hemoglobin. It is a major public health problem, particularly in the Mediterranean region, Middle East, and Southeast Asia. While blood transfusions and iron chelation therapy have improved the life expectancy of patients with thalassemia, they are not curative and have significant limitations. Cell-based gene therapy is a promising approach for the treatment of thalassemia. One such therapy, Zynteglo, involves the ex vivo transduction of autologous hematopoietic stem cells with a lentiviral vector carrying a functional copy of the β-globin gene. The modified cells are then infused back into the patient's bloodstream, where they differentiate into red blood cells that produce functional hemoglobin. This review provides an overview of thalassemia and the current state of cell-based gene therapy for this condition, with a focus on Zynteglo. It discusses the safety profile of the therapy, as well as its potential limitations. Keywords: Thalassemia, hemoglobin, gene therapy, zynteglo, prevention