Periodic Reporting for period 2 - DITSB (Development of Innovative Therapeutic Strategies for beta-hemoglobinopathies)

Beta-thalassemia and sickle cell disease (SCD) are caused by mutations affecting the synthesis or the structure of the adult hemoglobin (Hb) beta-chain. The only definitive cure is transplantation of allogeneic hematopoietic stem cells (HSCs) from a compatible donor, an option...