Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation.

ABSTRACT: Hemoglobinopathies are among the most common monogenic diseases worldwide. Approximately 1-5% of the global population are carriers for a genetic thalassemia mutation. The thalassemias are characterized by autosomal recessive inherited defects in the production of hemoglobin. They are highly prevalent in the Mediterranean, Middle East, Indian subcontinent, and East and Southeast Asia. Due to recent migrations, however, the thalassemias are now becoming more common in Europe and North America, making this disease a global health concern. Currently available conventional therapies in thalassemia have many challenges and limitations. A better understanding of the pathophysiology of β-thalassemia in addition to key developments in optimizing transfusion programs and iron-chelation therapy has

SUBMITTER: Motta I 

PROVIDER: S-EPMC7299245 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature