Progress and current status of gene therapy for thalassemia

Thalassemia is a hereditary hemolytic disease caused by the deficiency production of either the α-or β-globin chains, resulting from mutation or deletion of the α-or β-globin genes.Allogeneic hematopoietic stem cell transplantation is a curative option available for transfusion dependent thalassemia (TDT) patients.However, due to the limitation of matching donor sources, as well as the risk of unmatched donor transplantation, only fewer than 10% of patients are actually able to avail of the treatment.Gene therapy of autologous hematopoietic stem cell transplantation offers a new curative option for patients with TDT.Currently, there are two main strategies for gene therapy of β-thalassemia, one is through lentiviral vector mediated replacement of β-globin gene, and the other is through gene editing to re-active the expression of fetal γ-globin.There have been no reports about gene therapy for α-thalassemia until now.This article reviews the progress and status of gene therapy for β-thalassemia at home and abroad, and discusses the current problems and possible future directions of follow-up research of gene therapy for β-thalassemia.