beta-thalassemia is characterized by reduced or absence of beta-globin
production, resulting in anemia. Current therapies include blood
transfusion combined with iron chelation. BM transplantation, although
curative, is restricted by the matched donor limitation. Gene therapy,
on the other hand, is promising, and its success lies primarily on
designing efficient globin vectors that can effectively and stably
transduce HSCs. The major breakthrough in beta-thalassemia gene therapy
occurred a decade ago with the development of globin LVs. Since then,
researchers focused on designing efficient and safe vectors, which can
successfully deliver the therapeutic transgene, demonstrating no
insertional mutagenesis. Furthermore, as human HSCs have intrinsic
barriers to HIV-1 infection, attention is drawn towards their ex vivo
manipulation, aiming to achieve higher yield of genetically modified
HSCs. This paper presents the current status of gene therapy for
beta-thalassemia, its success and limitations, and the novel promising
strategies available involving the therapeutic role of HSCs
