Sickle cell disease and β-thalassemia represent the most common forms of hemoglobinopathies. Since the first successful bone marrow transplant in 1981, hematopoietic stem cell transplantation is the only treatment option for the patients. However, the current routine therapies for these conditions are limited by the availability of suitable donors and graft-vs-host disease. On the other hand, hemoglobinopathies were long considered amenable to the genetic correction by available gene transfer

more »

... chnologies. Therefore, gene therapy strategies aim at the globin gene transfer resulting hemoglobin function recovery. Here we review the studies and clinical applications of gene therapy for the hemoglobinopathies within the timeline of developed technologies, including the viral vectors, transposons, homolog recombination as a treatment modality together with generation of induced pluripotent stem cells and chimeraplasty. This brief review highlights the gene therapy strategies, current developments, challenges and future perspectives for hemoglobinopathies.