Advances in gene therapy for the treatment of sickle cell anemia

Currently, sickle cell anemia is a β-hemoglobinopathy, meaning a genetic autosomal recessive disease, affecting 400,000 newborns worldwide. Approximately 5% of the global population are carriers of genes causing this pathology. Regarding treatment, there are medical and pharmacological therapies for the disease; however, the only known curative option is allogeneic hematopoietic stem cell transplantation. Nonetheless, there is a high rejection rate, compounded by the fact that it is only available to a limited number of patients. Now, there is a new alternative: gene therapy, which is based on the addition and modification of genes.