NOVEL THERAPY FOR TREATMENT OF HEMOGLOBINOPATHIES (BETA-THALASSEMIA AND SICKLE CELL DISEASE)

Thalassemias are the result of the most common single gene genetic disorder worldwide, affecting 30 out of every 1000 people. Spanning from mild to life-threatening manifestations due to its autosomal recessive genetic inheritance, beta-thalassemia is caused by mutations in the y-globin gene. This can develop into decreased or complete loss of beta-globin expression. As a treatment of this disorder, increased levels of y-globin are pharmacologically induced to effectively compensate the absence of beta-globin chains. However, current treatments need to be improved, since they do not have the same positive outcome