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 few-er 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.
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维普
