Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia

The paper from Shrestha and colleagues1 focuses on the safety of gene therapy lentiviral vectors (LVs) to treat sickle cell anemia (SCA), a disease associated with the production of abnormal hemoglobin and red blood cells (RBCs), which can sickle and obstruct the blood flow, causing severe pain, organ damage, and reduced life expectancy. Current treatments have limitations, including hydroxyurea and allogeneic hematopoietic stem cell transplantation (allo-HSCT). Hydroxyurea’s effectiveness varies, and allo-HSCT is restricted by graft rejection and the scarcity of matched donors, underscoring the need for alternative therapies like gene therapy.