β-thalassemia is a genetic disorder resulting from defects in the β-globin
gene. Patients having a compatible human leukocyte antigen (HLA) matched donor can
be cured by transplantation of allogeneic hematopoietic stem cells (HSCs). However,
some recipients have a high risk of morbidity/mortality due to graft versus host disease
(GVHD) or graft rejection. Importantly, most patients do not have such HLA matchrelated
donor issues. Thus, the infusion of autologous HSCs modified with a lentiviral
vector expressing the β-globin therapeutic gene in the erythroid progenitors is a
promising approach to fully cure the disease. Here we review the history of β-
thalassemia treatment, particularly the development of the β-globin lentiviral vector,
with emphasis on clinical applications and future perspectives in a new era of
medicine.
Keywords: Gene Therapy, Hematopoietic Stem Cells, Precise Treatment,
Thalassemia.
