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Sickle cell disease is an autosomal recessive blood disorder that can lead to anaemia. It is caused by a mutation in the haemoglobin gene, which leads to deformation of red blood cells. Deformed red blood cells can obstruct small vessels and they are prone to destruction.
Sickle cell disease is a blood disorder that originates from a single point mutation in the HBB gene that codes for hemoglobin. Here, Moiani et al. developed an efficient TALEN-mediated HBB correction process that is compatible with gene therapy applications.
Sickle cell disease (SCD) and β-thalassemia (BT) are globally prevalent inherited blood disorders but, despite extensive research, no ex vivo system exists for SCD and BT. Here, the authors generate pathophysiologically relevant erythroid progenitor models of SCD and BT.