Advances in transplantation and gene therapy in transfusion-dependent β-thalassemia

Abstract

Transfusion-dependent thalassemia occurs globally and represents a major growing health problem worldwide. In majority, the disorder involves deficient or absent synthesis of the β-globin chains that constitute hemoglobin molecules and results in chronic hemolytic anemia. Subjects with the disorder must adhere to continuous red blood cell replacement program to sustain life; unfortunately such approach comes with undesirable and life-threatening complications. Without regular transfusions, thalassemic patients are prone to develop skeleton deformities, hepatosplenomegaly, and iron overload. Allogeneic hematopoietic cell transplantation preferably from HLA-matched sibling donor is widely accepted curative therapy. Gene therapy, although at its infancy, is emerging as an alternate curative option, however, with its own unique challenges. This article aims to review advances, challenges, controversies, and future prospects of allogeneic hematopoietic cell transplantation and gene therapy in subjects with transfusion dependent β-thalassemia.